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

Pathogenic mutations in mitochondrial DNA cause severe and often lethal multi-system symptoms in primary mitochondrial defects. However, effective therapies for these defects are still lacking. Strategies such as employing mitochondrially targeted restriction enzymes or programmable nucleases to shi...

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Detalles Bibliográficos
Autores principales: Chen, Xiaoxu, Chen, Mingyue, Zhu, Yuqing, Sun, Haifeng, Wang, Yue, Xie, Yuan, Ji, Lianfu, Wang, Cheng, Hu, Zhibin, Guo, Xuejiang, Xu, Zhengfeng, Zhang, Jun, Yang, Shiwei, Liang, Dong, Shen, Bin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10624837/
https://www.ncbi.nlm.nih.gov/pubmed/37923818
http://dx.doi.org/10.1038/s42003-023-05500-y

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