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    Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction

    Gene editing for the cure of inborn errors of metabolism (IEMs) has been limited by inefficiency of adult hepatocyte targeting. Here, we demonstrate that in utero CRISPR/Cas9-mediated gene editing in a mouse mode...

    Gourish Mondal, Caitlin J. VanLith, Clara T. Nicolas, Whitney S. Thompson… in Gene Therapy (2023)