Correction of a homoplasmic mitochondrial tRNA mutation in patient-derived iPSCs via a mitochondrial base editor

Chen, XX; Chen, MY; Zhu, YQ; Sun, HF; Wang, Y; Xie, Y; Ji, LF; Wang, C; Hu, ZB; Guo, XJ; Xu, ZF; Zhang, J; Yang, SW; Liang, D; Shen, B

Zhang, J; Shen, B (通讯作者),Nanjing Med Univ, State Key Lab Reprod Med & Offspring Hlth, Nanjing Matern & Child Hlth Care Hosp, Gusu Sch,Womens Hosp, Nanjing 211166, Peoples R China.;Xu, ZF; Liang, D (通讯作者),Nanjing Med Univ, Nanjing Matern & Child Hlth Care Hosp, Dept Prenatal Diag, Womens Hosp, Nanjing 210004, Peoples R China.;Yang, SW (通讯作者),Nanjing Med Univ, Childrens Hosp, Dept Cardiol, Nanjing 210008, Peoples R China.;Shen, B (通讯作者),Nanjing Med Univ, Sch Publ Hlth, Dept Epidemiol, Ctr Global Hlth, Nanjing 211166, Peoples R China.

COMMUNICATIONS BIOLOGY, 2023; 6 (1):

Abstract

Pathogenic mutations in mitochondrial DNA cause severe and often lethal multi-system symptoms in primary mitochondrial defects. However, effective the......

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