ARTICLE | Distillery Therapeutics
Therapeutics: Sarcoglycan γ (SGCG)
October 29, 2015 7:00 AM UTC
Insect, mouse and patient sample studies suggest exon skipping could help treat limb girdle muscular dystrophy type 2C (LGMD 2C), which is caused by frameshift mutations in exon 6 of SGCG. In an SGCG-...