Cas9-nickase–mediated genome editing corrects hereditary tyrosinemia in rats

Hereditary tyrosinemia type I (HTI) is a metabolic genetic disorder caused by mutation of fumarylacetoacetate hydrolase (FAH). Because of the accumulation of toxic metabolites, HTI causes severe liver cirrhosis, liver failure, and even hepatocellular carcinoma. HTI is an ideal model for gene therapy...

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Detalhes bibliográficos
Publicado no:J Biol Chem
Main Authors: Shao, Yanjiao, Wang, Liren, Guo, Nana, Wang, Shengfei, Yang, Lei, Li, Yajing, Wang, Mingsong, Yin, Shuming, Han, Honghui, Zeng, Li, Zhang, Ludi, Hui, Lijian, Ding, Qiurong, Zhang, Jiqin, Geng, Hongquan, Liu, Mingyao, Li, Dali
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Biochemistry and Molecular Biology 2018
Assuntos:
DNA and Chromosomes
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5936814/
https://ncbi.nlm.nih.gov/pubmed/29507093
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.RA117.000347
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