Adeno-associated viral delivery of engineered tRNA-enzyme pairs into nonsense mutation mouse models
Zhetao Zheng, Ningning Shi, Qing Xia
Journal：STAR Protocols
IF：0
DOI：10.1016/j.xpro.2022.101950
PMID：
Published：2022-12-16
research field：分子生物学基因治疗基因工程肌营养不良
Abstract
Summary In this protocol, we describe how to utilize the unnatural amino acid (UAA) incorporation system to read through endogenous premature termination codons in a Duchenne muscular dystrophy mouse model. We detail how to screen and optimize tRNA-enzyme pairs for efficient UAA incorporation, deliver the system intraperitoneally or intramuscularly in pathogenic mice by an adeno-associated viral (AAV) vector, and evaluate the restoration of endogenous dystrophin and increase in muscle strength after AAV injection. For complete details on the use and execution of this protocol, please refer to Shi et al. (2021).1
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