FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?

Himeda, Charis L; Jones, Peter L.

Himeda CL; The Department of Pharmacology, University of Nevada, Reno School of Medicine, Reno, NV 89557, USA.
Jones PL; The Department of Pharmacology, University of Nevada, Reno School of Medicine, Reno, NV 89557, USA.

J Pers Med ; 12(6)2022 May 25.

Article en En | MEDLINE | ID: mdl-35743650

Palabras clave

AAV; CRISPR; DUX4; FSHD; antisense; facioscapulohumeral muscular dystrophy; gene therapy; muscular dystrophy; skeletal muscle; therapeutics

Texto completo: 1 Banco de datos: MEDLINE Idioma: En Año: 2022 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Idioma: En Año: 2022 Tipo del documento: Article