Exon Skipping Therapy

Courtney S Young; April D Pyle

doi:10.1016/j.cell.2016.10.050

Exon Skipping Therapy

Cell. 2016 Nov 17;167(5):1144. doi: 10.1016/j.cell.2016.10.050.

Authors

Courtney S Young¹, April D Pyle²

Affiliations

¹ Molecular Biology Interdepartmental Program, Center for Duchenne Muscular Dystrophy, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA 90095.
² Molecular Biology Interdepartmental Program, Center for Duchenne Muscular Dystrophy, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA 90095; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095. Electronic address: apyle@mednet.ucla.edu.

PMID: 27863231
DOI: 10.1016/j.cell.2016.10.050

Abstract

Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using dystrophin expression as a surrogate marker. Exondys 51 targets DMD exon 51 for skipping to restore the reading frame for 13% of Duchenne patients.

Published by Elsevier Inc.

MeSH terms

Dystrophin / genetics*
Exons
Genetic Therapy*
Humans
Muscular Dystrophy, Duchenne / genetics*
Muscular Dystrophy, Duchenne / therapy*
Reading Frames
United States
United States Food and Drug Administration

Substances

Dystrophin