Anemia: progress in molecular mechanisms and therapies.
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Abstract | Anemia is a major source of morbidity and mortality worldwide. Here we review recent insights into how red blood cells (RBCs) are produced, the pathogenic mechanisms underlying various forms of anemia, and novel therapies derived from these findings. It is likely that these new insights, mainly arising from basic scientific studies, will contribute immensely to both the understanding of frequently debilitating forms of anemia and the ability to treat affected patients. Major worldwide diseases that are likely to benefit from new advances include the hemoglobinopathies (β-thalassemia and sickle cell disease); rare genetic disorders of RBC production; and anemias associated with chronic kidney disease, inflammation, and cancer. Promising new approaches to treatment include drugs that target recently defined pathways in RBC production, iron metabolism, and fetal globin-family gene expression, as well as gene therapies that use improved viral vectors and newly developed genome editing technologies. |
Year of Publication | 2015
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Journal | Nat Med
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Volume | 21
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Issue | 3
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Pages | 221-30
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Date Published | 2015 Mar
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ISSN | 1546-170X
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URL | |
DOI | 10.1038/nm.3814
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PubMed ID | 25742458
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PubMed Central ID | PMC4452951
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Grant list | R01 DK092318 / DK / NIDDK NIH HHS / United States
U01 HL117720 / HL / NHLBI NIH HHS / United States
R01 DK61692 / DK / NIDDK NIH HHS / United States
R01 DK061692 / DK / NIDDK NIH HHS / United States
R01 HL088554 / HL / NHLBI NIH HHS / United States
R01 DK103794 / DK / NIDDK NIH HHS / United States
R21 HL120791 / HL / NHLBI NIH HHS / United States
R01 DK0923128 / DK / NIDDK NIH HHS / United States
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