GenomicsbioRxivPreprint
Enhancement of Therapeutic Transgene Insertion for Murine Phenylketonuria
Michael Martinez, Daelyn Y. Richards, Shelley R. Winn…·Cary O. Harding
This study improves HDR-mediated gene insertion in mice with PAH deficiency by inhibiting competing DNA repair pathways, achieving transgene integration in 10% of alleles and reducing serum phenylalanine by 70.6%.
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