The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1
针对毒性 RNA 的 Cas9 持续表达可逆转 1 型强直性肌营养不良症小鼠模型中的疾病表型
期刊:Nature Biomedical Engineering
影响因子:26.8
doi:10.1038/s41551-020-00607-7
Ranjan Batra #, David A Nelles #, Daniela M Roth, Florian Krach, Curtis A Nutter, Takahiro Tadokoro, James D Thomas, Łukasz J Sznajder, Steven M Blue, Haydee L Gutierrez, Patrick Liu, Stefan Aigner, Oleksandr Platoshyn, Atsushi Miyanohara, Martin Marsala, Maurice S Swanson, Gene W Yeo
