A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice

A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice

Base editing tools enabled efficient conversion of C:G or A:T base pairs to T:A or G:C, which are especially powerful for targeting monogenic lesions. However, in vivo correction of disease-causing mutations is still less efficient because of the large size of base editors. Here, we designed a dual...

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Bibliographic Details
Main Authors: Lifang Zhou, Jing Su, Jie Long, Rui Tao, Wenling Tang, Fengming Qin, Nan Liu, Yanhong Wang, Yaoge Jiao, Yun Hu, Lurong Jiang, Li Li, Yang Yang, Shaohua Yao
Format: Article
Language:English
Published: Elsevier 2022-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
CRISPR/Cas9
Base editing
AAV
Gene therapy
Phenylketonuria
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012200002X

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http://www.sciencedirect.com/science/article/pii/S232905012200002X

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