Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD

• Main Authors: Ugur Akpulat (Author), Haicui Wang (Author), Kerstin Becker (Author), Adriana Contreras (Author), Terence A. Partridge (Author), James S. Novak (Author), Sebahattin Cirak (Author)
• Format: Book
• Published: Elsevier, 2018-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Duchenne muscular dystrophy is a fatal muscle disease, caused by mutations in DMD, leading to loss of dystrophin expression. Phosphorodiamidate morpholino splice-switching oligonucleotides (PMO-SSOs) have been used to elicit the restoration of a partially functional truncated dystrophin by excluding...