Bridging genomic discovery to potentially life-saving therapies, the key to treating the untreatable
Antisense therapies (antisense oligonucleotides or ASOs) are designed to bind precisely with RNA, halting the process of creating a disease-causing protein — disrupting diseases and changing their course—from the rarest of conditions, to those that impact millions of people. ASO’s are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of an ASO medicine is that they can be developed rapidly, inexpensively, and are highly specific.
Antisense Drugs Target the Messenger, Not the Protein Product
We Can Do This!
The n-Lorem Foundation and its partners are ready to evaluate patients with rare genetic diseases who may benefit from being treated with a custom experimental ASO. Here are two examples…
A child who experienced progressive loss of vision, epilepsy and neurological deterioration was found to have a unique mutation in a gene caused a variation of Batten disease. The child has been treated with an experimental ASO medicine and the disease may be progressing less rapidly.
A child born with an inability to feel pain experienced progressive deuteriation in vision and was found to have a unique genetic mutation that caused posterior column ataxia and retinitis pigmentosa (PCARP) and an experimental ASO medicine is being developed for the patient to try to prevent further vision loss and preserve the young patient’s ability to walk.