The primary objective of the Pediatric Hematology Research Training Program, which will complete its 35th year of funding in 2011, has been to prepare individuals interested in pediatric hematology for careers in academic medicine and biomedical research.
Specific aims i nclude (1) the provision of research training opportunities in a wide variety of basic and clinical research areas including hemoglobinopathies, megakaryocytes and platelets., immunohematology, stem cell development, molecular diagnostic techniques, molecular basis of hemostasis, and gene therapy for hematologic disorders;(2) the opportunity for structured coursework in cell and molecular biology, immunology, biochemistry, gene transfer, biostatistics, epidemiology and research design;(3) careful monitoring of the progress of individual trainees and the overall success of the training program;and (4) an emphasis on skills and accomplishments critical for successful academic careers such as preparation of manuscripts, presentations at seminars and national meetings, and submission of grant applications. The training faculty consists of a closely-knit group of physicians and scientists who have strong records as mentors and productive investigators, who have competed successfully for NIH and other federal funding, and who have interacted for many years in training and research activities. Extended research training is offered in a fellowship program designed to develop independent, funded investigators. Training opportunities have been enhanced by the setting with state-of-the-art research facilities, the recruitment of new, outstanding faculty members, and the awarding and/or renewal of program project grants in the areas of coagulation biology, gene transfer for hemophilia, and a Center for Excellence for Hematopoiesis. The program has emphasized the research training of minority students at all levels in order to increase the numbers of these individuals in biomedical careers. Despite a national trend away from pediatric subspecialty research training and toward clinical practice, the Pediatric Hematology Research Training Program continues to attract outstanding candidates and to prepare them for careers as biomedical investigators in academic institutions.
Researchers in pediatric hematology play an important role in the diagnosis and treatment of common inherited disorders including sickle cell disease, thalassemia, and hemophilia. Better treatments for these diseases will improve the quality of life of affected individuals and will reduce admissions to children's hospitals, where 50% of all admissions are for inherited diseases such as these.
|Gollomp, Kandace; Kim, Minna; Johnston, Ian et al. (2018) Neutrophil accumulation and NET release contribute to thrombosis in HIT. JCI Insight 3:|
|Greineder, Colin F; Johnston, Ian H; Villa, Carlos H et al. (2017) ICAM-1-targeted thrombomodulin mitigates tissue factor-driven inflammatory thrombosis in a human endothelialized microfluidic model. Blood Adv 1:1452-1465|
|George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam et al. (2017) Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med 377:2215-2227|
|Russell, Stephen; Bennett, Jean; Wellman, Jennifer A et al. (2017) Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet 390:849-860|
|Oved, Joseph H; Lee, Christina S Y; Bussel, James B (2017) Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone. J Pediatr 191:225-231|
|Arruda, Valder R; Doshi, Bhavya S; Samelson-Jones, Benjamin J (2017) Novel approaches to hemophilia therapy: successes and challenges. Blood 130:2251-2256|
|Bdeir, Khalil; Gollomp, Kandace; Stasiak, Marta et al. (2017) Platelet-Specific Chemokines Contribute to the Pathogenesis of Acute Lung Injury. Am J Respir Cell Mol Biol 56:261-270|
|Gollomp, Kandace; Lambert, Michele P; Poncz, Mortimer (2017) Current status of blood 'pharming': megakaryoctye transfusions as a source of platelets. Curr Opin Hematol 24:565-571|
|Nguyen, G N; George, L A; Siner, J I et al. (2017) Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A. J Thromb Haemost 15:110-121|
|George, Lindsey A (2017) Hemophilia gene therapy comes of age. Blood Adv 1:2591-2599|
Showing the most recent 10 out of 47 publications