Article

<i>In vivo</i> dissection of the mouse tyrosine catabolic pathway with CRISPR-Cas9 identifies modifier genes affecting hereditary tyrosinemia type 1

2023-09-29

Abstract excerpt

<h4>ABSTRACT</h4> Hereditary tyrosinemia type 1 is an autosomal recessive disorder caused by mutations (pathogenic variants) in fumarylacetoacetate hydrolase, an enzyme involved in tyrosine degradation. Its loss results in the accumulation of toxic metabolites that mainly affect the liver and kidneys and can lead to severe liver disease and liver cancer. Tyrosinemia type 1 has a global prevalence of approximately...

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Literature Corpus work: 03f24829-a56b-5d7d-87ff-561d4335bf52
DOI: 10.1101/2023.09.29.559947

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