Article

EX VIVO GENE EDITING AND CELL THERAPY FOR HEREDITARY TYROSINEMIA TYPE 1

2023-09-05

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background & Aims</h4> We previously demonstrated the successful use of in vivo CRISPR gene editing to delete 4-hydroxyphenylpyruvate dioxygenase ( HPD ) to rescue mice deficient in fumarylacetoacetate hydrolase (FAH), a disorder known as hereditary tyrosinemia type 1 (HT1). The goal of this study was to develop an ex vivo gene editing protocol and apply it as a cell therapy for HT1. <h...

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Literature Corpus work: 2f66f308-491d-5c7f-8729-1e2714ff49d9
DOI: 10.1101/2023.09.04.555940

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