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Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy.
Nat Commun. 2021 Dec 8;12(1):7128. doi: 10.1038/s41467-021-27430-1.
Nat Commun. 2021.
PMID: 34880230
Free PMC article.
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
Giesige CR, Wallace LM, Heller KN, Eidahl JO, Saad NY, Fowler AM, Pyne NK, Al-Kharsan M, Rashnonejad A, Chermahini GA, Domire JS, Mukweyi D, Garwick-Coppens SE, Guckes SM, McLaughlin KJ, Meyer K, Rodino-Klapac LR, Harper SQ.
Giesige CR, et al. Among authors: chermahini ga.
JCI Insight. 2018 Nov 15;3(22):e123538. doi: 10.1172/jci.insight.123538.
JCI Insight. 2018.
PMID: 30429376
Free PMC article.
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Large-Scale Production of Adeno-Associated Viral Vector Serotype-9 Carrying the Human Survival Motor Neuron Gene.
Rashnonejad A, Chermahini GA, Li S, Ozkinay F, Gao G.
Rashnonejad A, et al. Among authors: chermahini ga.
Mol Biotechnol. 2016 Jan;58(1):30-6. doi: 10.1007/s12033-015-9899-5.
Mol Biotechnol. 2016.
PMID: 26607476
Free PMC article.
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