CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice
利用 CRISPR/Cas9 技术靶向 Ube3a 反义转录本可改善小鼠的安格曼综合征表型。
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI142574
Schmid, Ralf S; Deng, Xuefeng; Panikker, Priyalakshmi; Msackyi, Msema; Breton, Camilo; Wilson, James M
