GEO series
DUX4.6 siRNA in vitro activity in FSHD patient-derived myotubes
GSE291267
Homo sapiens
Expression profiling by high throughput sequencing
32 samples
2026/03/08
GPL24676
Summary
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscular disease caused by the aberrant ectopic expression of DUX4 in skeletal muscle. There are no approved therapies for FSHD to date, and strategies aimed at reducing DUX4 expression in skeletal muscle of FSHD patients are promising therapeutic approaches. Here we demonstrate DUX4.6 siRNA activity in reducing DUX4-regulated gene expression in FSHD patient-derived myotubes in vitro.
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Paper (PMID 41994867) ↗
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