A Center of Molecular HematologyDescription of Major Goals
Support a core facility that can isolate rare hematopoietic populations and chromatin analysis for center users.
Development of novel selective Rac inhibitors for refractory leukemiasDescription of Major Goals
The goal of this project is to carry out orthogonal approaches to develop small molecule inhibitors of Rac for the treatment of refractory leukemias (in particular, MLL-rearranged and RAS-mutated).
FA PPG Core D: Preclinical Studies of Marrow Failure and Clonal Evolution in Fanconi AnemiaDescription of Major Goals
Evaluate the effects of small molecule compounds, metformin and TGF-beta receptor kinase inhibitors (TRKI), that improve proliferation, hematopoiesis, and cell survival in Fanconi anemia (FA) models, on the growth and differentiation of primary FA bone marrow, to investigate the effects of these compounds on DNA repair and clonal disease, and to develop a clinical trial to treat marrow failure in FA.