Seminar SeriesBeyond Compassionate Use: Building a Master Protocol to Advance Development of Individualized Medicines at Scale
PublicationsHow to create personalized gene editing platforms: Next steps toward interventional genetics
PublicationsScreening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study
Seminar SeriesDeciphering SPTAN1-associated disease mechanism and potential therapy in patient-derived cell lines
