Mark L. Batshaw
Developmental and Behavioral Pediatrics | Pediatrics
7125 13th Place Northwest
Washington, DC 20012
Washington, DC 20012
Last Updated: 09/26/2026
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Found 15 publications
Pediatric SARS-CoV-2 long term outcomes study (PECOS): cross sectional analysis at baseline.
Journal: Pediatric research
Published: June 07, 2024
The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study.
Journal: Translational science of rare diseases
Published: May 23, 2022
A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency.
Journal: Science advances
Published: April 03, 2019
Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders-A successful strategy for clinical research of rare diseases.
Journal: Journal of inherited metabolic disease
Published: February 12, 2019
Intellectual and developmental disabilities research centers: Fifty years of scientific accomplishments.
Journal: Annals of neurology
Published: December 14, 2018
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
Journal: Molecular genetics and metabolism
Published: January 12, 2017
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
Journal: Nature biotechnology
Published: September 23, 2015
A longitudinal study of urea cycle disorders.
Journal: Molecular genetics and metabolism
Published: July 31, 2014
Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer.
Journal: Human gene therapy
Published: September 10, 2013
Last Updated: 09/26/2026