Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I-Reference-Cited by-同舟云学术

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

Published:2021-06 Issue: Volume:21 Page:530-547
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

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^ORCID

Funder

Jiangmen Science and Technology Bureau

Guangdong Provincial Department of Science and Technology

International Science and Technology Cooperation Programme

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference70 articles.

1. On the enzymic defects in hereditary tyrosinemia;Lindblad;Proc. Natl. Acad. Sci. USA,1977

2. [Genetic counseling: experience of 4 years];Gagné;Union Med. Can.,1978

3. Geographical and ethnic distribution of mutations of the fumarylacetoacetate hydrolase gene in hereditary tyrosinemia type 1;Angileri;JIMD Rep.,2015

4. Fumarylacetoacetate, the metabolite accumulating in hereditary tyrosinemia, activates the ERK pathway and induces mitotic abnormalities and genomic instability;Jorquera;Hum. Mol. Genet.,2001

5. Fumarylacetoacetate inhibits the initial step of the base excision repair pathway: implication for the pathogenesis of tyrosinemia type I;Bliksrud;J. Inherit. Metab. Dis.,2013

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1. In vivo dissection of the mouse tyrosine catabolic pathway with CRISPR-Cas9 identifies modifier genes affecting hereditary tyrosinemia type 1;GENETICS;2024-08-23

2. Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment;Advanced Functional Materials;2024-05-27

3. Ex vivo gene editing and cell therapy for hereditary tyrosinemia type 1;Hepatology Communications;2024-04-26

4. An engineered Escherichia coli Nissle strain prevents lethal liver injury in a mouse model of tyrosinemia type 1;Journal of Hepatology;2023-11

5. In vivodissection of the mouse tyrosine catabolic pathway with CRISPR-Cas9 identifies modifier genes affecting hereditary tyrosinemia type 1;2023-09-29

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