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Open AccessAdvances in Sickle Cell Therapies in the Hydroxyurea Era
In the hydroxyurea era, insights into mechanisms downstream of erythrocyte sickling have led to new therapeutic approaches for patients with sickle cell disease (SCD). Therapies have been developed that target...
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Musings on genome medicine: Hepatitis C
Hepatitis C is a viral disease transmitted principally by blood, which affects millions of people worldwide. A significant proportion of those affected develop severe liver disease as a result. Only a fraction...
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Musings on genome medicine: enzyme-replacement therapy of the lysosomal storage diseases
The lysosomal storage diseases, such as Gaucher's disease, mucopolysaccharidosis I, II and IV, Fabry's disease, and Pompe's disease, are rare inherited disorders whose symptoms result from enzyme deficiency ca...
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Musings on genome medicine: Crohn's disease
The inflammatory bowel diseases, Crohn's disease and ulcerative colitis, pose a fascinating challenge to specialists in gastroenterology, infectious diseases, immunology and genetics and an often crushing burd...
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Musings on genome medicine: the slow but inexorable process of medical care reform in the United States
The current healthcare system in the United States is unsustainable, but any attempts at improvement must be carefully managed to avoid weakening the country's contribution to biomedical science research and t...
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Musings on genome medicine: the Obama effect redux
From the point of view of genome medicine, Barack Obama has made two vital policy decisions: he has chosen a new director of the National Institutes of Health, and his proposed change in United States healthca...
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Musings on genome medicine: cholesterol and coronary artery disease
Cholesterol levels and not inflammatory markers are the major variables that pose a risk of coronary artery disease. Diabetes greatly increases the risk at any cholesterol level. Coronary artery disease and ca...
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Musings on genome medicine: cancer genetics and the promise of effective treatment
Cancer is the most common acquired genetic disease. Great progress has been made in documenting the genetic abnormalities that cause the disease, and in the future each tumor will be subjected to genetic analy...
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Musings on genome medicine: gene therapy
Though the field has moved with glacial speed, gene therapies have been carried out successfully in patients with bone marrow disorders including immune deficiencies. The field may be poised to move forward mo...
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Musings on genome medicine: the Obama effect
The inauguration of the Obama administration is likely to enhance the role of genome medicine in clinical care and national economics.
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Musings on genome medicine: abuse of genetic tests
The wide general publication of a putative genetic test for athletic supremacy is clearly an abuse of genetics and reveals an undercurrent of hucksterism in biomedical science.
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Musings on genome medicine: genome wide association studies
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Fred S. Rosen (1930–2005)
Immunologist, paediatrician and polymath
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Acceptance of the 2003 John Howland Award: A Journey in Clinical Research
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The National Institutes of Health and clinical research: a progress report
The development of clinical investigators remains a thorny national problem. The National Institutes of Health has instituted new granting programs to support clinical research that have already stimulated 450...
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The βc Component of the Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)/Interleukin 3 (IL-3)/IL-5 Receptor Interacts with a Hybrid GM-CSF/Erythropoietin Receptor to Influence Proliferation and β-Globin mRNA Expression
The interaction of different members of the hematopoietic growth factor receptor family may be relevant to the increased proliferation and the failure of differentiation that characterizes the myeloid leukemia...
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A revived opportunity for fetal research
One of President Bill Clinton's early decisions was to lift the ban on federally supported research on human fetal tissue. Under what circumstances can research on this material be justified?
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Regulation of Hematopoiesis
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Summary and Conclusions
The management of aplastic anemia continues to challenge clinical investigators because the pathophysiology of the aplastic and hypoplastic syndromes is heterogeneous and poorly understood. The field can be fu...
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THE REGULATION OF HUMAN ERYTHROPOIESIS BY GROWTH FACTORS