HIGHLIGHTS
- who: Linde F. Bouwman from the Leiden University Washington, Seattle, WA, USA have published the research work: Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy, in the Journal: (JOURNAL) of 26/Dec/2021
- what: The authors show that the DUX4 ASO was well tolerated and repressed the DUX4 transcript DUX4 protein and mouse DUX4 target gene expression in skeletal muscles. This study shows that systemic delivery of ASOs targeting DUX4 is a promising therapeutic strategy for FSHD and strategies that further improve the ASO efficacy in skeletal muscle are warranted . . .
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