Article

Enhancement of Therapeutic Transgene Insertion for Murine Phenylketonuria

2025-02-28

Abstract excerpt

Low in vivo transgene integration frequency limits the therapeutic efficacy of homology-directed repair (HDR)-mediated gene insertion as a treatment for Mendelian disorders. This study demonstrates improved efficacy of HDR-mediated gene insertion for the treatment of murine phenylalanine hydroxylase (PAH) deficiency, a model of human phenylketonuria (PKU), through pharmacologic inhibition of competing DNA repair...

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Literature Corpus work: 5876e193-8dc0-55a7-90fd-7364ac00ff31
DOI: 10.1101/2025.02.24.639678

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