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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
JCI Insight. 2018 Nov 15;3(22):e123538. doi: 10.1172/jci.insight.123538.
JCI Insight. 2018.
PMID: 30429376
Free PMC article.
Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy.
Saad NY, Al-Kharsan M, Garwick-Coppens SE, Chermahini GA, Harper MA, Palo A, Boudreau RL, Harper SQ.
Saad NY, et al. Among authors: al kharsan m.
Nat Commun. 2021 Dec 8;12(1):7128. doi: 10.1038/s41467-021-27430-1.
Nat Commun. 2021.
PMID: 34880230
Free PMC article.
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