Gene therapy for Duchenne muscular dystrophy: an update on the latest clinical developments

  • Published on 11/17/2023
  •  Reading time: 4 min.

Cedric Happi Mbakam a,b & Jacques P. Tremblay a,b

a CHU de Québec research centre, Laval University, Québec, Canada
b Molecular Medicine Department, Faculty of Medicine, Laval University, Québec, Canada

CONTACT Jacques P. Tremblay mailto:Jacques-P.Tremblay@crchul.ulaval.ca CHU de Québec research centre, Laval University, Québec, Canada

Abstract

Introduction Duchenne muscular dystrophy (DMD) is one of the most severe and devastating neuromuscular hereditary diseases with a male newborn incidence of 20 000 cases each year. The disease caused by mutations (exon deletions, nonsense mutations, intra-exonic insertions or deletions, exon duplications, splice site defects, and deep intronic mutations) in the DMD gene, progressively leads to muscle wasting and loss of ambulation. This situation is painful for both...

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