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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

W. Wang,P. Zhou,X. Wang,F. Chen,E. Christensen,J. Thompson,X. Ren,A. Kells,L. Stanek,T. A. Carter,J. Hou,D. W. Y. Sah

Human Gene Therapy（2021）

引用 4|浏览28
关键词
RNAi,artificial microRNA,Huntington's disease,huntingtin lowering,in vivo
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