Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I

Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I

Hereditary tyrosinemia type I (HT1) results from the loss of fumarylacetoacetate hydrolase (FAH) activity and can lead to lethal liver injury (LLI). Therapeutic options for HT1 remain limited. The FAH−/− pig, a well-characterized animal model of HT1, represents a promising candidate for testing nove...

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Bibliographic Details
Main Authors: Peng Gu, Qin Yang, Bangzhu Chen, Ya-nan Bie, Wen Liu, Yuguang Tian, Hongquan Luo, Tao Xu, Chunjin Liang, Xing Ye, Yan Liu, Xiangwu Tang, Weiwang Gu
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
HPD ablation
tyrosinemia type I
lethal liver injury
pig
metabolic reprogramming
oxidative stress
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000681

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http://www.sciencedirect.com/science/article/pii/S2329050121000681

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