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The Children's Research Institute

Center for Genetic Medicine Research

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Genetic Medicine Research

Researching, diagnosing and treating genetic disorders.

Vision Statement

To transform children’s health through genome-enabled research, pre-clinical studies of experimental therapeutics, and clinical trials.

Mission

To understand health and disease as complex interactive processes, and to use this knowledge to restore health and prevent illness in childhood and throughout the lifespan.

The Center for Genetic Medicine Research

The center houses a highly interdisciplinary faculty, with nearly half of the physician-scientists from many clinical divisions in the hospital. Focusing on common health problems in Washington, DC, as well as serving as an international referral site for rare disorders, faculty and their laboratories are encouraged to be collaborative, and many of the Center’s projects bring together multiple clinical and scientific disciplines. The Center strives to provide faculty easy access to the latest technologies in genomics, proteomics, microscopy, bioinformatics, pre-clinical (murine) drug trials, and multi-site clinical trial networks. The Center provides services in these technologies to laboratories throughout the District, and internationally, through a series of NIH Core grants. Drug development and experimental therapeutics has increasingly become the focus, resulting in a technology transfer to an early-stage biopharmaceutical company, ReveraGen BioPharma, Inc.

We use data from interdisciplinary studies of molecules and pathways to create interactive physiological models of human health and disease that provide novel insights about potential strategies for disease prevention and treatment.

Related Links

Youssef A. Kousa, MS, D.O., Ph.D., a clinical fellow in the Division of Child Neurology at Children's National, explains how mutations in interferon regulatory factor 6 ( IRF6) that cause cleft lip and palate also are implicated in neural tube defects such as spina bifida.