Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.
利用靶向泛HTT的CRISPR核酸酶治疗亨廷顿病。
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doi:10.64898/2025.12.20.695720
Tan Katherine, Siller Daniela Del Bosque, Xiong Alisha Y, Wang Anna X A, McCallister Tristan X, Mummadi Sreya, St John Luke A, Lee Tae Kyu, Carrillo Annika, Renshaw Daniel G, Zhou Richard H, Lim Colin K W, He Jack, Fields Christopher J, Hayden Michael R, Gaj Thomas