Seminar SeriesTowards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders
Workshops & PresentationsNavigating Institutional Implementation: A Quarterly Open Forum for N-of-1 Clinical Trials
PublicationsConsensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
Seminar SeriesPersonalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
Seminar SeriesProject Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
