The applicant's overall commitment is to an academic research career in clinical investigative hematology, primarily devoted to the improvement of the care of patients with hemoglobinopathies, including those with sickle cell disease and the thalassemias. His long-term goals are to acquire the research skills and experience needed to become an independent investigator able to carry out the clinical studies that will be needed to make available to patients the new diagnostic technologies and therapeutic interventions now in development, including gene therapy, the manipulation of fetal hemoglobin production, stem-cell transplantation and other advances. With the support of the K23 Mentored Patient-Oriented Research Career Development Award, the applicant's immediate objectives are (i) completion of a two-year program of didactic exercises leading to a Master's Degree in Patient-Oriented Research from the School of Public Health of Columbia University, (ii) mentored involvement in the clinical studies required for the development of new methods for the assessment and management of transfusional iron overload in patients with hemoglobinopathies, and (iii) mentored participation as the leader of the Columbia University component of the Thalassemia Clinical Research Network. In order to achieve these objectives, the applicant will draw upon the vast resources for clinical research and training that are available at the Columbia University College of Physicians and Surgeons. All clinical research activity will be carried out through the NIH sponsored GCRC at the New York Presbyterian Hospital. The ultimate objectives are to enable the applicant to gain the training and expertise to be able to develop into an independent clinical investigator with a focus on patient-oriented research, in the setting of an academic institution.
