Seminar SeriesTowards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders
Seminar SeriesPersonalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder
Seminar SeriesThe Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
