Seminar SeriesPotential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic
Seminar SeriesSelecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
Seminar SeriesFrom Mutation to Dose-able CRISPR Medicine In 3 Months: Obstacles & Actionable Paths Forward
Seminar SeriesPerspectives on Clinical Outcomes for N=1 Patients: Insights from N1C Clinical Outcomes Workgroup
Seminar SeriesTailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
PublicationsSuppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
