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  1. No Access

    Article

    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)

  2. Article

    Open Access

    In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

    Conventional therapy for hereditary tyrosinemia type-1 (HT1) with 2-(2-nitro-4-trifluoromethylbenzoyl)−1,3-cyclohexanedione (NTBC) delays and in some cases fails to prevent disease progression to liver fibrosi...

    Clara T. Nicolas, Caitlin J. VanLith, Raymond D. Hickey, Zeji Du… in Nature Communications (2022)

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