CRISPR/Cas9-mediated correction of MITF homozygous point mutation in a Waardenburg syndrome 2A pig model

Gene therapy for curing congenital human diseases is promising, but the feasibility and safety need to be further evaluated. In this study, based on a pig model that carries the c.740T>C (L247S) mutation in MITF with an inheritance pattern and clinical pathology that mimics Waardenburg syndrome 2...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Nucleic Acids
Prif Awduron: Yao, Jing, Wang, Yu, Cao, Chunwei, Song, Ruigao, Bi, Dengfeng, Zhang, Hongyong, Li, Yongshun, Qin, Guosong, Hou, Naipeng, Zhang, Nan, Zhang, Jin, Guo, Weiwei, Yang, Shiming, Wang, Yanfang, Zhao, Jianguo
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2021
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC8141604/
https://ncbi.nlm.nih.gov/pubmed/34094716
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2021.04.009
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