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

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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Bibliographic Details
Main Authors: Camila F. Almeida, Florence Robriquet, Tatyana A. Vetter, Nianyuan Huang, Reid Neinast, Lumariz Hernandez-Rosario, Dhanarajan Rajakumar, W. David Arnold, Kim L. McBride, Kevin M. Flanigan, Robert B. Weiss, Nicolas Wein
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-06-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
aav
U7snRNA
myotonic dystrophy
gene therapy
spliceopathy
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2023.1181040/full

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