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CRISPR/Cas Applications in Myotonic Dystrophy: Expanding Opportunities

CRISPR/Cas technology holds promise for the development of therapies to treat inherited diseases. Myotonic dystrophy type 1 (DM1) is a severe neuromuscular disorder with a variable multisystemic character for which no cure is yet available. Here, we review CRISPR/Cas-mediated approaches that target...

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Detalles Bibliográficos
Autores principales:	Raaijmakers, Renée H.L., Ripken, Lise, Ausems, C. Rosanne M., Wansink, Derick G.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2019
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6696057/ https://www.ncbi.nlm.nih.gov/pubmed/31357652 http://dx.doi.org/10.3390/ijms20153689

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