Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy

Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is caused by chromatin relaxation of the D4Z4 repeat resulting in misexpression of the D4Z4-encoded DUX4 gene in skeletal muscle. One of the key genetic requirements for the stable production of full-length DUX4 mRNA in skeletal muscle is a functional po...

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Bibliographic Details
Main Authors: Darina Šikrová, Vlad A. Cadar, Yavuz Ariyurek, Jeroen F.J. Laros, Judit Balog, Silvère M. van der Maarel
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
facioscapulohumeral muscular dystrophy
DUX4
polyadenylation signal
CRISPR-Cas9
base editing
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121001360

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