Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates
在体外以及在小鼠和非人类灵长类动物中对亨廷顿蛋白降低腺相关病毒基因治疗进行优化的主要人工微小 RNA 的有效和精确处理
期刊:Human Gene Therapy
影响因子:3.9
doi:10.1089/hum.2021.221
Wei Wang, Pengcheng Zhou, Xin Wang, Fen Chen, Emily Christensen, Jeffrey Thompson, Xiaoqin Ren, Adrian Kells, Lisa Stanek, Todd Carter, Jay Hou, Dinah W Y Sah
