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Research Summaries in Hematologic Diseases
Jeffery L. Miller, MD: Genome-Based Studies of Erythroid Biology and Disease
Dr. Miller is focused upon understanding the molecular and genetic basis of red cell development and toward finding treatments for diseases involving those cells. Relationships between erythroblast growth, signal transduction, and the regulation of erythroid-specific genes are being explored. Clinical projects include novel approaches toward comparing the clinical phenotype of patients with erythroid diseases with genome- or proteome-based descriptions of their blood. The laboratory provides state-of-the-art equipment and informatics. For more information see our database at http://hembase.niddk.nih.gov or search PUBMED using "miller jl bethesda".
John F. Tisdale, MD: Hematopoietic Stem Cell Based Approaches to Disorders of Globin Synthesis
Hematologic disorders such as the thalassemias and hemoglobinopathies, resulting from absent/reduced or abnormal production of one or more of the globin-molecule subunits, respectively, together constitute the most prevalent group of human monogenic diseases. Strategies which aim to replace the absent or defective globin gene have long been envisioned as potentially curative, and our group is focused on the use of hematopoietic stem cells to achieve this goal. We have recently developed a low intensity conditioning regimen designed to promote tolerance to allogeneic hematopoietic stem cells for adults with thalassemia and sickle cell anemia, and are actively accruing patients. For those lacking a suitable donor, the development of therapeutic approaches based on genetic manipulation of autologous hematopoietic stem cells remains a viable alternative. We are currently working in small and large animals to perform preclinical, translational studies in stem cell gene transfer and transplantation to maximize both safety and potential efficacy prior to clinical application.
Last updated:
12/14/2006 |
