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Excel · study · 2026

DUX4.6 siRNA in vitro activity in FSHD patient-derived myotubes

Listed in NCBI GEO

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscular disease caused by the aberrant ectopic expression of DUX4 in skeletal muscle.

Description

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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Expression profiling by high throughput sequencing · Homo sapiens
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Life Sciences
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Disease 75%
Provenance · 1 source records, 8 field assertions
SourceKeyLast seenRaw
NCBI GEOGSE29126715 d agoJSON v1
FieldAssertionExtractorEvidence
access_levelsource · NCBI GEOconnector:ncbi_geo@1.0.0
concepts[disease].local:disease:diseaseenrichment · NCBI GEOkeyword-concept-rules@1.0.0title+description (75%)
concepts[field].local:field:life-sciencesmapping · NCBI GEOconnector:ncbi_geo@1.0.0
concepts[method].geo_series_type:expression-profiling-by-high-throughput-sequencingsource · NCBI GEOconnector:ncbi_geo@1.0.0/gdstype
concepts[organism].NCBITaxon:9606source · NCBI GEOconnector:ncbi_geo@1.0.0/taxon
descriptionsource · NCBI GEOconnector:ncbi_geo@1.0.0/summary
publication_datesource · NCBI GEOconnector:ncbi_geo@1.0.0
titlesource · NCBI GEOconnector:ncbi_geo@1.0.0/title