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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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Detalles Bibliográficos
Autores principales:	Šikrová, Darina, Cadar, Vlad A., Ariyurek, Yavuz, Laros, Jeroen F.J., Balog, Judit, van der Maarel, Silvère M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8399085/ https://www.ncbi.nlm.nih.gov/pubmed/34484861 http://dx.doi.org/10.1016/j.omtn.2021.05.020

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