Lesson 17 – The Science of ASO Design and Screening Using the FDA Guidance for Nano-rare Patients
Lessons in Antisense [...]
Hope for Kinsley: Facing TUBB4A with Courage and Care
with Sarah and Ryan Marta
Lesson 16 – PS ASO-induced PS ASO-RNP Aggregates
Lessons in Antisense [...]
Lesson 15 – The Mechanisms of PS ASO-induced Cytotoxicity
Lessons in Antisense [...]
New Insights into Genetically Caused Developmental Delays
Emerging [...]
Splice-modulating antisense oligonucleotides targeting a pathogenic intronic variant in adult polyglucosan body disease correct mis-splicing and restore enzyme activity in patient cells
Thomas, R., Miyoshi, E., Akman, H.O., Storz, S.H.R., Goffena, J., Pytte, J., Miller, D.E., Skourti-Stathaki, K., Crooke, S.T.
Lesson 14 – PS ASO Cellular Uptake and Distribution
Lessons in Antisense [...]
Lesson 13 – PS ASOs and Innate Immunity
Lessons in Antisense [...]
