Articles & Podcasts'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis
Seminar SeriesSplice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa
PublicationsThe N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing
Seminar SeriesPreclinical development of personalized ASO therapeutics using patient-derived organoid systems
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
