A non-myeloablative conditioning regimen for hematopoietic stem cell transplantat

Abstract Hematopoietic stem cell transplantation (HSCT) has become a standard care for the treatment of many hematologic malignancies and non-malignant diseases such as bone marrow failure and immunodeficiency syndromes. Currently, myeloablative or irradiative conditioning regimens are used for enhancing HSC engraftment in transplantation, but they are often associated with significant morbidity and mortality, particularly in patients under severe clinical or pathological stress. In addition, in many clinical cases only limited numbers of HSCs are available for transplant. Improving efficiency of BM niche access will improve HSCT outcomes by increasing donor chimerism in clinical settings where stem cell numbers are limiting and recipients are fragile, e.g.in cord blood or gene therapy transplants. As an important intracellular signal transducer of multiple cell stimuli required for HSC maintenance, including signaling from receptor tyrosine kinase c-Kit, chemokine receptor CXCR4 and adhesion receptor integ