Development of a high-throughput screen to identify small molecule enhancers of sarcospan for the treatment of Duchenne muscular dystrophy.
Cynthia ShuAriana N Kaxon-RuppJudd R ColladoRobert DamoiseauxRachelle H CrosbiePublished in: Skeletal muscle (2019)
We developed a stable muscle cell line containing the promoter region of the human SSPN protein fused to a fluorescent reporter. Using the reporter cells, we created and validated a scalable, cell-based assay that is able to identify compounds that increase SSPN promoter reporter, transcript, and protein levels in wild-type and dystrophin-deficient muscle cells.
Keyphrases
- duchenne muscular dystrophy
- high throughput
- induced apoptosis
- small molecule
- wild type
- crispr cas
- cell cycle arrest
- single cell
- dna methylation
- skeletal muscle
- transcription factor
- gene expression
- endothelial cells
- rna seq
- cell death
- endoplasmic reticulum stress
- signaling pathway
- binding protein
- oxidative stress
- living cells
- amino acid
- bone marrow
- pi k akt
- single molecule