This application is for a competitive renewal of a multi-categorical General Clinical Research Center locatedat the Children's Hospital of Cincinnati. The Center provides inpatient, outpatient and scatter bed facilitieswithin the Children's Hospital and a Core Laboratory supporting biochemical assays, body compositionmeasurements, and behavioral studies at the Children's Hospital. A proposed satellite at the VeteransAdministration Medical Center will help support studies in medically unstable adults who are not suitable tobe studied at the Children's Hospital GCRC. Major areas of research include:1. Cardiovascular disease: Studies are defining the potential role of differences in cholesterol absorption oncholesterol metabolism, the effects of disease states such as hypertension and chronic renal disease inchildren on cardiac and vascular function and anatomy.2. Behavioral Interventions: Studies are exploring the effect of behavioral intervention in enhancing nutrientintake in cystic fibrosis, sickle cell disease, Crohn's Disease and others and assessing the impact of earlytraumatic brain injury on later neurocognitive development.3. Cancer: Novel investigations are ongoing evaluating the effect of rapammune and similar compounds forthe treatment of angiolipomata associated with tuberous sclerosis and lymphangioleiomyomatosis (LAMS)for whom no effective therapy other than surgery is available.4. Obesity: Studies are defining the roles of changes in body composition in early life on the development oflater obesity, examining racial disparities in body composition which occur during adolescence, and theimpact of obesity on sleep disturbances and carbohydrate metabolism.5. Gene Transfer: Gene transfer trials have been initiated at the GCRC to evaluate whether insertion of awild type gene into the stem cells of patients with Fanconi Anemia will be safe and ultimately whether it willbe efficacious in correcting their underlying bone marrow defects.6. Diabetes Mellitus: The impact of Type 2 diabetes and obesity on cardiac and vascular structure andfunction in adolescents is being evaluated. The role of the insulinotropic Gl hormone glucagon-like peptide 1(GLP-1) in gastric emptying and insulin secretion in normal and Type 2 diabetics is being examined and itsrole in type 2 diabetes.7. Inborn errors of bile acid metabolism: With a long track record of research and discovery of multipleenzymatic defects in the bile acid synthetic pathways, investigators at Children's Hospital continue toenhance understanding of these defects affecting infants and children with cholestasis and fat solublevitamin deficiencies.
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