Enhancement of Therapeutic Transgene Insertion for Murine Phenylketonuria
Michael Martinez, Daelyn Y. Richards, Shelley R. Winn, Adrian M. Baris, Anne Vonada, Sandra Dudley, Laura Harper, 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%.
bioRxiv · Feb 24, 2025 · PreprintGenomics