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There should be, and in the best of cases there is, a synergy between basic research and patient care. However, this synergy is hard to develop because the techniques required to be a successful researcher are so different from the skills required to be an outstanding physician. Harold R. Roberts, M.D., of the University of North Carolina at Chapel Hill, is an example of a physician-researcher who has benefited from having his feet in both the world of patient care and the world of the laboratory: he has let clinical problems direct his basic research effort and conversely has adopted research advances in his care of patients. Dr. Roberts's long and continuing career has included many research and clinical advances. He was part of the first group to determine the amino acid sequence of the important thrombin inhibitor hirudin and part of the group that prepared the first cryoprecipitates which were the first alternative to plasma as therapy in hemophilia A. Dr. Roberts has made significant advances in understanding the protein chemistry behind hemophilia B; he was among the first researchers to identify some patients as not being completely deficient but instead as having measurable levels of protein and subsequently demonstrated that this protein was dysfunctional. This important advance led him to a classification scheme for patients into Cross Reacting Material (CRM) positive, negative, and reduced. Dr.
List of contents
I. Infectious Disease Risk.- 1. The Transfusion-Transmitted Viruses in Blood Transfusion.- II. Unexplained Aspects of Hemophilia and Hemostasis.- 2. Are Patients with Glanzmann Thrombasthenia and the Bernard-Soulier Syndrome Protected Against Atherosclerosis?.- 3. Factor V: Dr. Jeckyll and Mr. Hyde.- III. Novel Approaches to Therapy.- 4. Gene Therapy for Hemophilia B: AA V-Mediated Transfer of the Gene for Coagulation Factor IX to Human Muscle.- 5. Use of Prophylaxis to Prevent Complications of Hemophilia.- 6. Factor VIII Inhibitors.- 7. Use of High Dose Factor VIIa in Hemophilia Patients.- IV. Inhibitor Development in Hemophilia Patients.- 8. The Incidence of Inhibitors in Hemophilia A and the Induction of Immune Tolerance.- 9. Mechanisms of B-Cell Tolerance.- 10. Mechanisms of Unresponsiveness: T- and B-Cell Mediated Mechanisms of Anergy.- 11. CD4+ T Cells Specific for Factor VIII as a Target for Specific Suppression of Inhibitor Production.- 12. CD40L Pathway Blockade as an Approach to Immuno-therapy.- Abstracts.- Contributors.
Summary
There should be, and in the best of cases there is, a synergy between basic research and patient care. However, this synergy is hard to develop because the techniques required to be a successful researcher are so different from the skills required to be an outstanding physician. Harold R. Roberts, M.D., of the University of North Carolina at Chapel Hill, is an example of a physician-researcher who has benefited from having his feet in both the world of patient care and the world of the laboratory: he has let clinical problems direct his basic research effort and conversely has adopted research advances in his care of patients. Dr. Roberts's long and continuing career has included many research and clinical advances. He was part of the first group to determine the amino acid sequence of the important thrombin inhibitor hirudin and part of the group that prepared the first cryoprecipitates which were the first alternative to plasma as therapy in hemophilia A. Dr. Roberts has made significant advances in understanding the protein chemistry behind hemophilia B; he was among the first researchers to identify some patients as not being completely deficient but instead as having measurable levels of protein and subsequently demonstrated that this protein was dysfunctional. This important advance led him to a classification scheme for patients into Cross Reacting Material (CRM) positive, negative, and reduced. Dr.
