Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model

Aberrant expression of the double homeobox 4 (DUX4) gene in skeletal muscle causes muscle deterioration and weakness in Facioscapulohumeral muscular dystrophy (FSHD). Since the presence of a permissive pLAM1 polyadenylation signal is essential for stabilization of DUX4 mRNA and translation of DUX4 p...

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Detalles Bibliográficos
Autores principales: Lu-Nguyen, Ngoc, Malerba, Alberto, Herath, Shan, Dickson, George, Popplewell, Linda
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
General Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8283208/
https://www.ncbi.nlm.nih.gov/pubmed/33987655
http://dx.doi.org/10.1093/hmg/ddab136

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8283208/
https://www.ncbi.nlm.nih.gov/pubmed/33987655
http://dx.doi.org/10.1093/hmg/ddab136

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