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Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression

Silencing the expression of the double homeobox 4 (DUX4) gene offers great potential for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Several research groups have recently reported promising results using systemic antisense therapy in a transgenic small animal model of FSHD, the A...

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Detalles Bibliográficos
Autores principales: Lu-Nguyen, Ngoc, Dickson, George, Malerba, Alberto, Popplewell, Linda
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9313434/
https://www.ncbi.nlm.nih.gov/pubmed/35884928
http://dx.doi.org/10.3390/biomedicines10071623