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Office
111 Michigan Ave Nw
Washington, DC 20010Phone+1 202-884-5467
Education & Training
- Johns Hopkins UniversityResidency, Pediatrics, 1973 - 1975
- University of Chicago Division of the Biological Sciences The Pritzker School of MedicineClass of 1971
Certifications & Licensure
- CA State Medical License 1973 - Present
- MD State Medical License 1974 - 2026
- VA State Medical License 2022 - 2026
- DC State Medical License 1998 - 2024
- PA State Medical License 1987 - 2000
- NJ State Medical License 1989 - 1999
- American Board of Pediatrics Pediatrics
- American Board of Psychiatry and Neurology Neurodevelopmental Disabilities
Awards, Honors, & Recognition
- Whos Who in Medicine and Healthcare Marquis Whos Who
Clinical Trials
- Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency Start of enrollment: 1995 Oct 01
- Longitudinal Study of Urea Cycle Disorders Start of enrollment: 2006 Feb 01
Publications & Presentations
PubMed
- 47 citationsA mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency.Lili Wang, Yang Yang, Camilo Breton, Peter Bell, Mingyao Li
Science Advances. 2020-02-01 - 394 citationsA dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn miceYang Yang, Lili Wang, Peter Bell, Deirdre McMenamin, Zhenning He
Nature Biotechnology. 2016-03-01 - 35 citationsTransatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders-a successful strategy for clinical research of rare diseasesRoland Posset, Sven F. Garbade, Nikolas Boy, Alberto Burlina, Carlo Dionisi-Vici
Journal of Inherited Metabolic Disease. 2018-07-04
Press Mentions
- Children’s National Gets Millions in Federal Funding for Pandemic PreparednessJuly 19th, 2022
- The Quest to Overcome Gene Therapy’s FailuresOctober 18th, 2021
- Nobel Laureate, NIH Leaders to Present 2021-22 Maury Strauss Public LecturesAugust 5th, 2021
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Grant Support
- Child Health Research Career Development AwardEunice Kennedy Shriver National Institute Of Child Health &Human Development2002–2012
- Training (Career Development) ComponentEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2011
- Rdcrc Administrative UnitEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2011
- RARE Diseases Clinical Research Consortia (RDCRC) For The Rdcr NetworkEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2011
- RARE Diseases Clinical Research Consorita (RDCRC) For The Rdcr NetworkEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2011
- Longitudinal Study Of Urea Cycle DisordersEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2011
- Investigation Of Brain Nitrogen Metabolism In Partial Ornithine TrascarbamylaseEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2011
- General Clinical Research CenterNational Center For Research Resources2005–2010
- RARE Diseases Crc- Urea Cycle DisordersNational Center For Research Resources2004–2008
- RARE Diseases Crc: Urea Cycle DisordersNational Center For Research Resources2003–2008
- AdministrationEunice Kennedy Shriver National Institute Of Child Health &Human Development2006
- Mrddrc At Children'S National Medical CenterEunice Kennedy Shriver National Institute Of Child Health &Human Development2001–2006
- Pbtc 007v30 -A Phase 1/11trial Of ZD1839 (IRESSATM)National Center For Research Resources2005
- Developing &Improving Animal ResourcesNational Center For Research Resources2002
- Extramural Research Facilities ConstructionNational Center For Research Resources1999
- Human Studies Of Ornithine Transcarbamylase DeficiencyEunice Kennedy Shriver National Institute Of Child Health &Human Development1995–1999
- Gene Therapy For Ornithine Transcarbamylase DeficiencyEunice Kennedy Shriver National Institute Of Child Health &Human Development1995–1999
- Nichd Mental Retardation Research Center Core GrantEunice Kennedy Shriver National Institute Of Child Health &Human Development1990–1998
- Society For Inherited Metabolic Disorders SymposiumNational Institute Of Diabetes And Digestive And Kidney Diseases1996
- Neurotransmitters, Appetite &Inborn Metabolism ErrorsNational Institute Of Neurological Disorders And Stroke1989–1991
- Urea Cycle Disorders--Effects Of Neurotransmitters And BehaviorNational Center For Research Resources1990
- Alternate Pathway Therapy For Inborn Errors Of Urea SynthesisNational Center For Research Resources1990
- Neurotransmitters,Appetite,&Inborn Errors Of MetabolismNational Institute Of Mental Health1988
- Neurotransmitters,Appetite,&Inborn Errors Of MetabolismNational Institute Of Mental Health1986–1987
- Asymptomatic Hyperammonemia--A Cause Of Cortical DysfunctionEunice Kennedy Shriver National Institute Of Child Health &Human Development1985
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