PublicationsPossibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders
Seminar SeriesThe Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
Illustrated ExamplesConsensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs
PublicationsAntisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic later
