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A Systemically Administered Unconjugated Antisense Oligonucleotide Targeting DUX4 Improves Muscular Injury and Motor Function in FSHD Model Mice

Facioscapulohumeral muscular dystrophy (FSHD), one of the most common muscular dystrophies, is caused by an abnormal expression of the DUX4 gene in skeletal muscles, resulting in muscle weakness. In this study, we investigated MT-DUX4-ASO, a novel gapmer antisense oligonucleotide (ASO). MT-DUX4-ASO...

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Detalles Bibliográficos
Autores principales:	Kakimoto, Tetsuhiro, Ogasawara, Akira, Ishikawa, Kiyoshi, Kurita, Takashi, Yoshida, Kumiko, Harada, Shuichi, Nonaka, Taeko, Inoue, Yoshimi, Uchida, Keiko, Tateoka, Takashi, Ohta, Tetsuya, Kumagai, Shinji, Sasaki, Takashi, Aihara, Hajime
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10525656/ https://www.ncbi.nlm.nih.gov/pubmed/37760780 http://dx.doi.org/10.3390/biomedicines11092339

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