Mark L. Batshaw

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

Steven E. Raper, Narendra Chirmule, Frank S. Lee et al. · Molecular Genetics and Metabolism · 2003 · 1.5K citations

Inflammation, Autoimmune Disease, Disease Mechanism +9

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang Yang, Lili Wang, Peter Bell et al. · Nature Biotechnology · 2016 · 552 citations · Full text

Systems Biology, Hepatology, Cas9-mediated Correction +10

Neurologic Outcome in Children with Inborn Errors of Urea Synthesis

Michael E. Msall, Mark L. Batshaw, Richard A. Suss et al. · New England Journal of Medicine · 1984 · 411 citations

Neurodevelopmental Disorders, Intellectual Impairment, Developmental Anomaly +15

Clinical research for rare disease: Opportunities, challenges, and solutions

Robert C. Griggs, Mark L. Batshaw, Mary Dunkle et al. · Molecular Genetics and Metabolism · 2008 · 390 citations · Full text

Clinical Population, Clinical Studies, Clinical Trials +3

A Pilot Study of <i>In Vivo</i> Liver-Directed Gene Transfer with an Adenoviral Vector in Partial Ornithine Transcarbamylase Deficiency

Steven E. Raper, Marc Yudkoff, Narendra Chirmule et al. · Human Gene Therapy · 2002 · 350 citations