Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy in Vivo.
Jia LiKaiying WangYuchen ZhangTuan QiJuanjuan YuanLei ZhangHan QiuJinxi WangHuang-Tian YangYi DaiYan SongXing ChangPublished in: Circulation (2021)
Our study demonstrates the feasibility and efficacy to institute exon skipping through an enhanced TAM (eTAM) for therapeutic application(s).