CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy MAGIC WP3
收藏Duchenne Data Repository2026-08-02 收录
数据链接：
官方服务：
资源简介：
Publication on CRISPR-mediated upregulation of utrophin as a therapeutic strategy for Duchenne muscular dystrophy (DMD) through targeted mutation of the miRNA Let-7c binding site. The approach was validated in cell lines, engineered 3D skeletal muscle tissues, and in vivo using the mdx mouse model of DMD.
