Cincinnati Cooperative Center of Excellence in Hematology

SUMMARY/Abstract: The long-term goal of the Cincinnati Cooperative Center of Excellence in Hematology (CCCEH) is to understand and correct, at the molecular level, hematological diseases of various lineages. To attain this goal, we must understand basic biological processes that affect behaviors of hematopoietic stem cells and their

various differentiated progenies in vitro and in vivo. We envision this long-term goal will be best accomplished by interdisciplinary and collaborative approaches using state-of-the-art methods, along with timely and rational use of translational studies into the preclinical and clinical settings. Cincinnati Children's Hospital Medical

Center (CCHMC) has developed a rich and stimulating intellectual environment that brings together outstanding expertise and basic research in stem cell biology, virus vector technology, immunohematologic cell biology, genomics and genetics, and hematopoiesis; in addition, the CCCEH has world-class expertise in

translational and clinical research in hematology, immunology and hematopoietic stem cell transplantation. This focus has been greatly strengthened by the recruitment of over 30 faculty members in these areas of hematology research over the last 15-years at CCHMC, and the inclusion of over 15 external hematology

researchers around US. Basic research is linked with Cincinnati NIH-funded Clinical and Translational Science Award (CTSA), which has facilitated interdisciplinary interactions and developed an infrastructure that supports state-of-the-art clinical trials in cell and gene therapy. Here we seek to solidify the exciting and systemic growth

of research in hematology at CCHMC to support a national center of excellence, using the shared services and administrative structure of the center grant mechanism. The CCCEH Administrative Core supports an exceptional array of research cores, which function together to support innovative basic and translational

research both locally and nationally, in non-malignant hematology. Our three proposed research cores include (1) Xenotransplant and Genome Editing Core, which maintains specialized mouse strains and provides mouse transplant and transgenic services; (2) Single Cell Characterization and Procurement Core that includes state-

of-the-art imaging as well as single cell capture and gene expression capabilities; and (3) Gene Delivery Core that covers services such as ex vivo manipulation of blood cells by lentivirus and retrovirus transduction and shRNA/sgRNA screening. Finally, this center seeks support for an innovative Enrichment Program that fosters

a collaborative and educational environment in the center and for outreach to the broad hematology research community around US.