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2011 - NIH Clinical Center - National Institutes of Health

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RARE DISEASES CATEGORY: PROJECTS CO-FUNDED BY OFFICE OF RARE DISEASES RESEARCH AND INSTITUTES/CENTERSPROJECTTargeting Antigen-Antibody Responsesin Systemic Capillary Leak SyndromeSympathetic Innervation & MyocardialInjury in Acute Stress CardiomyopathyPreclinical Testing <strong>of</strong> Targeted Agentsfor <strong>Clinical</strong> Development in NF1The DICER1-related PleuropulmonaryBlastoma Cancer PredispositionSyndromeBrain Development in Children withWilliams Syndrome and theLIMK1 GeneThe Role <strong>of</strong> EGFR inEndolymphatic Sac TumorsINVESTIGATORS INSTITUTE(S)/INSTITUTIONNIAID: K. Druey; T. Myers; S. Porcella; NCI: O. Landgren; NIBIB: A. Gorbach;University <strong>of</strong> Minnesota: A. Dudek; Mayo College <strong>of</strong> Medicine: P. GreippCC (Radiology and Imaging Sciences): C. Sibley; D. Bluemke; NINDS: D. Goldstein;NHLBI: D. Rosing; Johns Hopkins: I. Wittstein; F. Bengel; J. Mudd; J. LimaNCI: A. Kim; B. Widemann; E. Dombi; Children’s Hospital Medical <strong>Center</strong>: N. Ratner; J. WuNCI: C. Kratz; B. Alter; P. Rosenberg; <strong>National</strong> Children’s Medical <strong>Center</strong>: A. Hill;Children’s Hospital & Clinics <strong>of</strong> MN: Y. Messinger; K. SchulzNIMH: K. Berman; J. Kleinman; University <strong>of</strong> Louisville: C. MervisNCI: P. Dennis; Yale University: A. VortmeyerRARE DISEASES DRUG DEVELOPMENT: PROJECTS CO-FUNDED BY THERAPEUTICS FOR RARE AND NEGLECTED DISEASES PROGRAMAND INSTITUTES/CENTERSA Novel Therapy to Treat Acid Lipase-Deficiency by LCAT InhibitionDevelopment <strong>of</strong> Combination Therapyfor Niemann-Pick Disease, Type CGene Therapy <strong>Clinical</strong> Trial for LAD-1Using a Foamy Viral VectorBH3 Mimetics for the Treatment <strong>of</strong>Autoimmune LymphoproliferativeSyndromePreventing Aortic Dilation in Womenwith Turner SyndromeImmunogenicity and LeishmaniaVaccine Potential <strong>of</strong> Sandfly Saliva inHumansNHLBI: A. Remaley; K. Vickers; R. Shamburek; Cincinnati Children’s Hospital Medical<strong>Center</strong> - Research Foundation: G. Grabowski; H. DuNICHD: F. Porter; A. Yergey; S. Bianconi; NHGRI: W. Pavan;Washington University: D. OryNCI: D. Hickstein; University <strong>of</strong> Washington Medical <strong>Center</strong>: D. Russell; CincinnatiChildren’s Hospital Medical <strong>Center</strong>: P. MalikCC (Laboratory Medicine): J. Oliveira Filho; T. Fleisher; NIAID: V. Rao; K. Dowdell;NIDDK: D. AppellaNICHD: C. Bondy; V. Bakalov; J. Zhou; NHLBI: A. Arai; D. Rosing; M. Boehm; V. Sachdev;Johns Hopkins University: J. Van Eyk; Q. FuNIAID: J. Valenzuela; S. Kamhawi; Uniformed Services University <strong>of</strong> the <strong>Health</strong> Sciences/Walter Reed Army Medical <strong>Center</strong>: N. Aronson; George Washington University:M. BottazziPROJECT FUNDED BY THE NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENTThe Role <strong>of</strong> BDNF in Autism SpectrumDisorder and Cognitive FunctionNICHD: J. Han; C. Pierpaoli; NIMH: K. Martinowich; D. Weinberger; C. Golden Williams;S. Swedo; A. Thurm; NCI: L. Tessarollo; Uniformed Services University <strong>of</strong> the <strong>Health</strong>Sciences/Walter Reed Army Medical <strong>Center</strong>: S. Sharp; GlaxoSmithKlinePharamceuticals: B. LuANNUAL REPORT <strong>2011</strong>• 25

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