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...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Veröffentlicht in:J Biol Chem
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Biochemistry and Molecular Biology 2018
Schlagworte:
DNA and Chromosomes
Online Zugang: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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