Duchenne muscular dystrophy: genome editing gives new hope for treatment

Crispi, Vassili; Matsakas, Antonios

Abstract

© Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted. Duchenne muscular dystrophy (DMD) is a progressive wasting disease of skeletal and cardiac muscles, representing one of the most common recessive fatal inherited genetic diseases with 1:3500-1:5000 in yearly incidence. It is caused by mutations in the DMD gene that encodes the membrane-associated dystrophin protein. Over the years, many have been the approaches to management of DMD, but despite all efforts, no effective treatment has yet been discovered. Hope for the development of potential therapeutics has followed the recent advances in genome editing and gene therapy. This review gives an overview to DMD and summarises current lines of evidence with regard to treatment and disease management alongside the appropriate considerations.

Citation

Crispi, V., & Matsakas, A. (2018). Duchenne muscular dystrophy: genome editing gives new hope for treatment. Postgraduate Medical Journal, 94(1111), 296-304. https://doi.org/10.1136/postgradmedj-2017-135377

• Journal Article Type: Review
• Acceptance Date: Jan 13, 2018
• Online Publication Date: Jan 31, 2018
• Publication Date: 2018-05
• Deposit Date: May 25, 2022
• Journal: Postgraduate medical journal
• Print ISSN: 0032-5473
• Electronic ISSN: 1469-0756
• Publisher: BMJ Publishing Group
• Peer Reviewed: Peer Reviewed
• Volume: 94
• Issue: 1111
• Pages: 296-304
• DOI: https://doi.org/10.1136/postgradmedj-2017-135377
• Public URL: https://hull-repository.worktribe.com/output/3608186