Our research focuses on a) the mechanisms by which blood cancers are resistant to drug treatments and how we can use all the information we can obtain, including mutations, gene expression, and functional testing results against a panel of drugs, to choose the best treatment for an individual patient; and b) gene therapy for an inherited blood disorder called Fanconi anemia. For cancer: We take blood and bone marrow samples from patients with blood cancers such as acute leukemia or multiple myeloma, analyze them for mutations known to be prevalent, analyze the expression of genes, and test them in a special laboratory here at ISCRM against a panel of >150 drugs and drug combinations. We then utilize this information to choose treatments for patients. We also study the biology of how the cells become cancerous, how they evolve over time, and test new drugs and methods to improve outcomes for the patients. For Fanconi anemia: We take patient stem cells derived from the bone marrow, treat them in the laboratory to provide a copy of the normal gene, and reinfuse the cells into the patient. The benefit to the patients will be to achieve a response to new treatment for their blood cancer and prolong their survival, or to improve blood counts in patients with Fanconi anemia who have been treated with gene therapy.