Submitted Genes
Highly-targeted
gene specific programs
A personalized mutation-driven drug discovery program
Our nano-rare patients are a very, very small patient population, often many will be the only one in the world with their specific gene mutation. As such, the majority of our patients have a unique-to-them drug discovery program in development.
Our genes are large and there can be many different mutations within a single gene. Each mutation can cause a very different outcome in terms of cause of disease, disease symptoms and severity. At n-Lorem, we not only target the specific gene, but design our program to address the specific gene mutation for each patient.
It is our hope that, as more genome sequencing is conducted, more patients will be identified that have a gene mutation that fits a program we are already working on. For those patients, we will already be well ahead in discovering and developing a potential experimental ASO medicine.
Submitted genes
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
While we cannot accept every application that is submitted to n-Lorem, we learn from each application and continue to advance our understanding of nano-rare patients and their unique illnesses.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
Nano-rare patients present a unique opportunity to learn about health and disease, and we are obligated to learn maximally from each patient and our aggregate experience and share our learnings with all interested parties as rapidly as possible.
Below is a list of all the genes that have been submitted to n-Lorem. This list is updated quarterly. In some instances, we have had multiple submissions for a single gene, each submission with different gene mutations.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
Genes from all submissions:
Not all are active programs
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
- NOTE:A maroon dot next to a gene indicates an available ASO therapy. Click on the gene name to learn more.
ASXL3
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ASXL3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ATN1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ATN1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
CHCHD10
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the CHCHD10 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
ELP1
- ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the ELP1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
FLVCR1
- Mutation
c.1193A>G; p.Tyr398Cys - ASO Strategy
Splicing modulation - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the FLVCR1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
GARS1
- Mutation
c.894_905del; p.Glu299_Gln302del - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the GARS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
H3F3A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the H3F3A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
HNRNPH2
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
KIF1A
- Mutation
c914C>T; pP305L - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the KIF1A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
LMNB1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a duplication in the LMNB1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
MAPK8IP3
- Mutation
c.1714C>T; p.Arg572Cys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PACS1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PACS1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
PRPH2
- Mutation
c.623G>A; p.Gly208Asp - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the PRPH2 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SCN2A
- Mutation
c.2558 G>A; p.Arg853Gln
c.5645 G>A; p.Arg1882Gln - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SCN2A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SERPINI1
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SERPINI1 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SAA1
- ASO Treatment Therapy
We have a potential therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver. This therapy has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TDP43
- Mutation
c.1135 T>C; p.Ser379Pro - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
TUBB4A
- ASO Strategy
Non-allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the TUBB4A gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
UBTF
- Mutation
c.628G>A; p.Glu210Lys - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the UBTF gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
SLC12A6
- Mutation
c.2971A>G; p.Thr991Ala - ASO Strategy
Allele-selective - ASO Treatment Therapy
We have a therapy available for a nano-rare patient with a mutation in the SLC12A6 gene that has the potential to help other patients with a mutation in this gene. If you are a patient or patient family or a physician of a potential patient, please email us at [email protected] for more information on this program.
- NOTE:A orange dot next to a gene indicates an active program. Click on the gene name to learn more.
ADCY5
- ASO Strategy
Allele-selective - Active ASO Program
ANXA11
- ASO Strategy
Allele-selective - Active ASO Program
ATL1
- ASO Strategy
Allele-selective - Active ASO Program
ATM
- ASO Strategy
Splicing modulation - Active ASO Program
CACNA1A
- ASO Strategy
Allele-selective - Active ASO Program
CACNA1E
- ASO Strategy
Allele-selective - Active ASO Program
CAMK4
- ASO Strategy
Allele-selective - Active ASO Program
CLCN7
- ASO Strategy
Allele-selective - Active ASO Program
DHDDS
- ASO Strategy
Allele-selective - Active ASO Program
DNAJC5
- ASO Strategy
Allele-selective - Active ASO Program
DNM1
- ASO Strategy
Allele-selective - Active ASO Program
DYRK1A
- ASO Strategy
Upregulation - Active ASO Program
EIF2AK2
- ASO Strategy
Allele-selective - Active ASO Program
FBXO31
- ASO Strategy
Allele-selective - Active ASO Program
GBE1
- ASO Strategy
Splicing modulation - Active ASO Program
GNAO1
- ASO Strategy
Allele-selective - Active ASO Program
GRIA1
- ASO Strategy
Allele-selective - Active ASO Program
GUCA1A
- ASO Strategy
Allele-selective - Active ASO Program
KCNB1
- ASO Strategy
Allele-selective - Active ASO Program
KCNH1
- ASO Strategy
Allele-selective - Active ASO Program
KCNQ2
- ASO Strategy
Allele-selective - Active ASO Program
KCNT2
- ASO Strategy
Non-allele-selective - Active ASO Program
NALCN
- ASO Strategy
Allele-selective - Active ASO Program
NARS1
- ASO Strategy
Allele-selective - Active ASO Program
NEFH
- ASO Strategy
Allele-selective - Active ASO Program
PACS2
- ASO Strategy
Allele-selective - Active ASO Program
PFN1
- ASO Strategy
Allele-selective - Active ASO Program
PLA2G6
- ASO Strategy
Splicing modulation - Active ASO Program
PLP1
- ASO Strategy
Splicing modulation - Active ASO Program
RHOBTB2
- ASO Strategy
Allele-selective - Active ASO Program
SAMD9L
- ASO Strategy
Allele-selective - Active ASO Program
SCN8A
- ASO Strategy
Splicing modulation - Active ASO Program
SETX
- ASO Strategy
Allele-selective - Active ASO Program
SLC37A4
- ASO Strategy
Allele-selective - Active ASO Program
SPTAN1
- ASO Strategy
Allele-selective - Active ASO Program
SPTLC1
- ASO Strategy
Allele-selective - Active ASO Program
CYFIP2
- ASO Strategy
Allele-selective - Active ASO Program
DHX30
- ASO Strategy
Allele-selective - Active ASO Program
POLR3B
- ASO Strategy
Splicing modulation - Active ASO Program
PRPF31
- ASO Strategy
Splicing modulation - Active ASO Program
RHOB
- Active ASO Program
RNU4-2
- ASO Strategy
Allele-selective - Active ASO Program
SPTSSA
- ASO Strategy
Allele-selective - Active ASO Program
TNPO2
- ASO Strategy
Allele-selective - Active ASO Program
ATXN3
- Active ASO Program
AUTS2
- ASO Strategy
Upregulation - Active ASO Program
CERT1
- Active ASO Program
DEAF1
- Active ASO Program
GRIA2
- Active ASO Program
SYT1
- Active ASO Program
- NOTE: A gray dot next to a gene name indicates a gene where there is no path forward for our ASO technology today.
BRAD-KIAA1549
- Out of scope
n-Lorem has evaluated this gene fusion, and today, there is no path forward for ASO technology to provide therapeutic benefit for patients with this gene fusion
ANO5
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
LMNA
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
RFC1
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
NAB2-STAT6
- Out of scope
n-Lorem has evaluated this gene fusion, and today, there is no path forward for ASO technology to provide therapeutic benefit for patients with this gene fusion
DES
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
PYROXD1
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
HSPB8
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
COL6A2
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
TTN
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
MTAP
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
MT-ND1
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
ADSSL1
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
SPAST
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
SMC1A
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
EPM2A
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
SETD1B
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
MEF2C
- Out of scope
n-Lorem has evaluated this gene, and today, there is no path forward for ASO technology to provide therapeutic benefit for mutations in this gene
- TAOK1
- TARDBP (TDP43)
- TCF4
- TECPR2
- THAP12
- TIMMDC1
- TNPO2
- TREX1
- TSC2
- TTN
- TUBB2A
- TUBB2B
- TUBB3
- TUBB4A
- TUBB4B
- YWHAG
What we cannot treat today:
- Gene expression is primarily in organs other than CNS, liver, kidney and eye
- Truncating variants
- True null or loss of function variants
- Homozygous gene mutation
However, ASO technology continues to advance at a rapid rate, and we hope to one day be able to broaden our efforts to include nano-rare patients who are not amenable to ASO technology today.
What do I do if my gene isn’t listed?
This is a list of all of the genes that have been submitted to n-Lorem. Not all are active programs. To learn more if you or your patient qualifies for n-Lorem please visit one of the following links:
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