
Mark Batshaw
Neurodevelopmental Pediatrician · George Washington University School of Medicine
Credentials: · · · ·
History
Research
Case report · Molecular Genetics and Metabolism · 2003 · Last of 8 authors · 1,081 citations
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Nature Biotechnology · 2016 · 449 citations
New England Journal of Medicine · 1984 · 319 citations

23 papers
- MY
10 papers
- MT
8 papers

7 papers

7 papers · 6 as a fellow

6 papers

5 papers
- JC
5 papers
Grants
- 2022–2027
Rare Disease Clinical Research Training Program
National Institute of Child Health and Human Development · R25
PI · $482.6K · Wake Forest University Health Sciences
- 2001–2020
Child Health Research Career Development Award (CHRCDA) Program at Children's National
National Institute of Child Health and Human Development · K12
Contact PI · $5.6M · Children's Research Institute
- 2003–2019
National Institute of Child Health and Human Development · U54
Contact PI · $11M · Children's Research Institute
- 2008–2018
Gene Therapy for Urea Cycle Disorders
National Institute of Child Health and Human Development · P01
PI · $5.5M · University of Pennsylvania
Clinical trials
- 1995
Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency
Study chair · Terminated · Phase 1
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

