Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy [Medical Sciences]

Duchenne muscular dystrophy (DMD) is primarily caused by out-of-frame deletions in the dystrophin gene. Exon skipping using phosphorodiamidate morpholino oligomers (PMOs) converts out-of-frame to in-frame mutations, producing partially functional dystrophin. Four single-exon skipping PMOs are approved for DMD but treat only 8 to 14% of patients each, and some exhibit...

Source: Proceedings of the National Academy of Sciences - February 22, 2022 Category: Science Authors: Kenji Rowel Q. Lim, Stanley Woo, Dyanna Melo, Yiqing Huang, Kasia Dzierlega, Md Nur Ahad Shah, Tejal Aslesh, Rohini Roy Roshmi, Yusuke Echigoya, Rika Maruyama, Hong M. Moulton, Toshifumi Yokota Tags: Medical Sciences Biological Sciences Source Type: research