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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.
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Submitted on : Friday, July 17, 2020 - 4:17:42 PM
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Océane Ballouhey, Marc Bartoli, Nicolas Levy. CRISP(R)ation musculaire. médecine/sciences, EDP Sciences, 2020, 36 (4), pp.358-366. ⟨10.1051/medsci/2020081⟩. ⟨hal-02901910⟩

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