Basic and Translational Research Training in Pediatric Classical Hematology

PROJECT SUMMARY Non-malignant blood disorders affect millions of individuals worldwide. Studies of these diseases and normal hematopoiesis (i.e. Classical Hematology) contribute to our general understanding of normal tissue development, mechanisms of gene expression, and medical genetics. Indeed, sickle cell disease, the first “molecular disease”, is the first disorder to be FDA-approved for therapeutic genome editing. More than 120 genes regulating virtually all fundamental cellular processes have been implicated in the mechanisms of inherited bone marrow failure (BMF) syndromes, and investigations of myeloproliferative neoplasms (MPNs) have advanced our understanding of cytokine receptor signaling. Despite these advances, non-malignant blood disorders remain a major health problem, warranting the development of new effective therapies. Progress towards this is hampered by a critical shortage of biomedical trainees focused on Classical Hematology. Our proposed training program addresses this critical shortage by attracting trainees to the field and equipping them with the excitement, knowledge, and skills to make new biological discoveries. St. Jude Children’s Research Hospital’s commitment to trainee education, focus on advancing cures for pediatric Classical Hematology disorders, and exceptional resources for biomedical research support this goal. Our program will support three postdoctoral fellows with enriched training in basic and translational Classical Hematology, focusing on hypothesis-driven research that aims to elucidate the mechanisms of non-malignant blood diseases and uncover new therapeutic targets. Trainees will employ state-of-the-art -omics technologies, genome engineering, in vitro and in vivo models of hematologic disease, and rigorous statistical testing to accomplish their research goals. The program is supported by faculty with diverse expertise, a mentorship committee for each trainee, an Internal Steering Committee to advise on program development and training activities, and an External Advisory Board comprised of five national leaders in Classical Hematology. Training elements include a dedicated lecture series, opportunities for relevant clinical exposure and education, a quarterly discussion series devoted to career development, a Training Day that includes trainee and invited speaker presentations, a grant writing workshop, and individualized mentoring committees and career development plans with detailed evaluation of trainee progress. This program will expand the pool of highly- qualified future leaders in laboratory-based Classical Hematology research.