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

• Main Authors: Jing Yao (Author), Yu Wang (Author), Chunwei Cao (Author), Ruigao Song (Author), Dengfeng Bi (Author), Hongyong Zhang (Author), Yongshun Li (Author), Guosong Qin (Author), Naipeng Hou (Author), Nan Zhang (Author), Jin Zhang (Author), Weiwei Guo (Author), Shiming Yang (Author), Yanfang Wang (Author), Jianguo Zhao (Author)
• Format: Book
• Published: Elsevier, 2021-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...