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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:J Biol Chem
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society for Biochemistry and Molecular Biology 2018
Gaiak:
DNA and Chromosomes
Sarrera elektronikoa: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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