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2015 ; 21
(3
): 221-30
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Anemia: progress in molecular mechanisms and therapies
#MMPMID25742458
Sankaran VG
; Weiss MJ
Nat Med
2015[Mar]; 21
(3
): 221-30
PMID25742458
show ga
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 (?-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.