Rare Diseases Bench-to-Bedside 2003 Awards 1. Investigation of Brain and Behavioral Effects on Specific Gene Deleted in the "Williams Syndrome Critical Area" of Chromosome 7q11.23 in patients and Knockout Mice. Principal Investigator(s)/Institute(s): Karen Faith Berman, M.D. (NIMH) Jacqueline Crawley, Ph.D. (NIMH) Abstract 2. Sialic Acid Replacement in Patients with Hereditary Inclusion Body Myopathy (HIBM) Caused by Mutations in the GNE (Sialuria) Gene. Principal Investigator(s)/Institute(s): Marinos C. Dalakas, M.D., Ph.D. (NINDS) Marjan Huizing, Ph.D. (NHGRI) Abstract 3. Dendritic Cell Vaccination to Augment Graft-versus-Leukemia after Allogenic Stem Cell Transplantation for Acute Lymphoblastic Leukemia (ALL). Principal Investigator(s)/Institute(s): Alan. S. Wayne, M.D. (NCI) Chrystal L. Mackall, M.D. (NCI) Abstract 4. Evaluation of HMG-Reductase Inhibitors in Pulmonary Sarcoidosis. Principal Investigator(s)/Institute(s): Matthew Park, M.D., Ph.D. (NHLBI) Vincent Manganiello, M.D., Ph.D. (NHLBI) Abstract 5. Clonal Insertion Site Analysis in Rhesus Macaques and NIH Patients Transplanted with Retrovirally Transduced Hematopoietic Progenitor and Stem Cells: Implications for Leukemogenesis. Principal Investigator(s)/Institute(s): Cynthia Dunbar, M.D. (NHLBI) Fabio Candotti, M.D., Ph.D. (NHGRI) Harry Malech, M.D. (NIAID) Abstract   Last Reviewed: November 09, 2007