Seminar SeriesSplice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa
Seminar SeriesTowards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders
Seminar SeriesConnecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Seminar SeriesSelecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
Seminar SeriesTailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
