Review Article

The advances and challenges of Gene Therapy for Duchenne Muscular Dystrophy

Jacques P Tremblay* and Jean-Paul Iyombe-Engembe

Published: 25 July, 2017 | Volume 1 - Issue 1 | Pages: 019-036

Since the discovery of the dystrophin gene (DMD gene) thirty years ago, several therapeutic approaches have been investigated to treat Duchenne muscular dystrophy (DMD). This includes cell therapy, exon jumping, exonic knockout, and the CinDel method. In this article, we present the challenges of developping a treatment for DMD and the advances of these various approaches. We included the new CRISPR-Cas9 system, which permits not only major progress in the development of new treatments based on genome editing but also the production of new animal models.

Read Full Article HTML DOI: 10.29328/journal.jgmgt.1001003 Cite this Article Read Full Article PDF


Gene therapy, Duchenne muscular dystrophy, CRISPR/Cas9, Animal model


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