Rescue of autosomal dominant hearing loss by in vivo delivery of mini dCas13X-derived RNA base editor

Xiao, QQ; Xu, ZJ; Xue, YY; Xu, CL; Han, L; Liu, YH; Wang, F; Zhang, RZ; Han, S; Wang, X; Li, GL; Li, HW; Yang, H; Shu, YL

Yang, H (通讯作者)，Chinese Acad Sci, CAS Ctr Excellence Brain Sci & Intelligence Techn, Shanghai Res Ctr Brain Sci & Brain Inspired Intel, Inst Neurosci,State Key Lab Neurosci,Key Lab Prim, Shanghai 200031, Peoples R China.;Li, HW; Shu, YL (通讯作者)，Fudan Univ, ENT Inst, Shanghai 200031, Peoples R China.;Li, HW; Shu, YL (通讯作者)，Fudan Univ, Dept Otorhinolaryngol, Eye & ENT Hosp, State Key Lab Med Neurobiol, Shanghai 200031, Peoples R China.;Li, HW; Shu, YL (通讯作者)，Fudan Univ, MOE Frontiers Ctr Brain Sci,

SCIENCE TRANSLATIONAL MEDICINE, 2022; 14 (654):

Abstract

Programmable RNA editing tools enable the reversible correction of mutant transcripts, reducing the potential risk associated with permanent genetic c......

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