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

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...

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Bibliographic Details
Main Authors: Jing Yao, Yu Wang, Chunwei Cao, Ruigao Song, Dengfeng Bi, Hongyong Zhang, Yongshun Li, Guosong Qin, Naipeng Hou, Nan Zhang, Jin Zhang, Weiwei Guo, Shiming Yang, Yanfang Wang, Jianguo Zhao
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
CRISPR/Cas9
base editor
MITF
gene correction
anophthalmia
hearing loss
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121001037

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