Leah E. Venturoni

Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

Randy J. Chandler, Leah E. Venturoni, Jing Liao et al. · Hepatology · 2020 · 53 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +17

New Cases of DHTKD1 Mutations in Patients with 2-Ketoadipic Aciduria

Ashlee R. Stiles, Leah E. Venturoni, Grace Mucci et al. · JIMD Reports · 2015 · 37 citations · Full text

Life Sciences, Genetics, Molecular Genetics +16

In vivo genome editing at the albumin locus to treat methylmalonic acidemia

Jessica L. Schneller, Ciaran M. Lee, Leah E. Venturoni et al. · Molecular Therapy — Methods & Clinical Development · 2021 · 19 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing

Leah E. Venturoni, Randy J. Chandler, Jing Liao et al. · Molecular Genetics and Metabolism · 2022 · 14 citations · Full text

Life Sciences, Genetics, Molecular Biology +17