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The Children's National Research Institute

Research Programs

We study common pediatric health problems as well as relatively rare inherited diseases. Our strong translational focus includes drug development programs, biomarker discovery, pre-clinical testing, genetic analysis, natural history studies to derive clinical outcome measures, and clinical trials. Below you will find more information regarding specific research areas:

Airway and Lung

Read about some Airway and Lung diseases that research is focused on in at the Center for Genetic Medicine Research.


Asthma is the most common chronic childhood disease and has become considerably more prevalent and severe in the United States during the last 40 years. The observed symptoms - wheezing, coughing, chest tightness and shortness of breath - are a result of an inflammatory response in the airways. Genetic, environmental and social risk factors for asthma have been established, but an exact cause is unknown.

Brain Injury and Disorders

The central nervous system group within the Center for Genetic Medicine Research works closely with investigators in the Center for Neuroscience Research and the Center for Cancer and Immunology Research. Key investigators are Susan Knoblach, Ph.D., on spinal cord trauma and ALS, Javad Nazarian, Ph.D., on pediatric brain tumors, and Yetrib Hathout, Ph.D., working on neurofibromatosis.

Intermediary Metabolism

This program explores the clinical effects in variation in basic life-sustaining biochemistry under conditions of genetic and/or  environmental disruption.

Kidney, Heart Disease, and Diabetes

Children’s National Research Institute is currently conducting research on pediatric kidney diseases including polycystic kidney disease.

Muscle and Muscular Dystrophy

Our interdisciplinary muscular dystrophy research and clinical programs are an internationally-recognized resource for researchers, patients/families and physicians.

Obesity and Type 2 Diabetes

Obesity and type 2 diabetes are epidemic health care problems that have both lifestyle (diet, inactivity) and genetic (familial risk) components. Metabolic syndrome is the presence of multiple risk factors for cardiometabolic disease that often leads to type 2 diabetes and cardiovascular disease. Greater than 85 percent of obese adolescents will become obese adults, contributing to escalating adult disease rates.

Urea Cycle Disorders (UCD)

Children’s National is considered the world leader in the diagnosis, treatment, and research of urea cycle disorders with three renowned experts in this field. Mendel Tuchman, MD, Mark Batshaw, MD, and Marshall Summar, MD, lead nation-wide research and clinical programs for these disorders.