Skip to Main content Skip to Navigation
New interface
Journal articles

CRISP(R)ation musculaire

Abstract : Muscular dystrophies are a group of rare muscular disorders characterized by weakness and progressive degeneration of the muscle. They are diseases of genetic origin caused by the mutation of one or more genes involved in muscle function. Despite significant progress made in the field of biotherapies in recent years, there is as yet no curative treatment available for these diseases. Studies conducted since the discovery of the CRISPR-Cas9 genomic editing tool have nevertheless led to significant and promising advances in the treatment of muscular dystrophies. CRISPR-Cas9 system allows a stable and permanent edition of the genome and should make it possible to avoid long, partially efficient and repetitive treatments. In this review, we will discuss the latest therapeutic advances obtained using the CRISPR-Cas9 system in genetic muscular dystrophies.
Complete list of metadata

Cited literature [50 references]  Display  Hide  Download
Contributor : Marc Bartoli Connect in order to contact the contributor
Submitted on : Friday, July 17, 2020 - 4:17:42 PM
Last modification on : Monday, February 14, 2022 - 10:20:12 AM
Long-term archiving on: : Tuesday, December 1, 2020 - 12:28:52 AM


Publisher files allowed on an open archive


Distributed under a Creative Commons Attribution 4.0 International License




Océane Ballouhey, Marc Bartoli, Nicolas Levy. CRISP(R)ation musculaire. médecine/sciences, 2020, 36 (4), pp.358-366. ⟨10.1051/medsci/2020081⟩. ⟨hal-02901910⟩



Record views


Files downloads