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Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults and affects mainly the skeletal muscle, heart, and brain. DM1 is caused by a CTG repeat expansion in the 3′UTR region of the DMPK gene that sequesters muscleblind-like proteins, blocking their splicing activity a...

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Detalles Bibliográficos
Autores principales:	Almeida, Camila F., Robriquet, Florence, Vetter, Tatyana A., Huang, Nianyuan, Neinast, Reid, Hernandez-Rosario, Lumariz, Rajakumar, Dhanarajan, Arnold, W. David, McBride, Kim L., Flanigan, Kevin M., Weiss, Robert B., Wein, Nicolas
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2023
Materias:	Cell and Developmental Biology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10309041/ https://www.ncbi.nlm.nih.gov/pubmed/37397246 http://dx.doi.org/10.3389/fcell.2023.1181040

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