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 no...

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Detalles Bibliográficos
Autores principales: Gu, Peng, Yang, Qin, Chen, Bangzhu, Bie, Ya-nan, Liu, Wen, Tian, Yuguang, Luo, Hongquan, Xu, Tao, Liang, Chunjin, Ye, Xing, Liu, Yan, Tang, Xiangwu, Gu, Weiwang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8099604/
https://www.ncbi.nlm.nih.gov/pubmed/33997102
http://dx.doi.org/10.1016/j.omtm.2021.04.002

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