期刊
LANCET
卷 387, 期 10037, 页码 2554-2564出版社
ELSEVIER SCIENCE INC
DOI: 10.1016/S0140-6736(15)01341-0
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资金
- Canadian Institute of Health Research (MOP) [123382]
- Canada Research Chair Program, National Institute of Diabetes and Digestive and Kidney Diseases [K08DK093705]
- Doris Duke Charitable Foundation
- Charles H Hood Foundation
- Cooley's Anemia Foundation
- Biogen
Sickle-cell disease affects millions of individuals worldwide, but the global incidence is concentrated in Africa. The burden of sickle-cell disease is expected to continue to rise over the coming decades, adding to stress on the health infrastructures of many countries. Although the molecular cause of sickle-cell disease has been known for more than half a century, treatment options remain greatly limited. Allogeneic haemopoietic stem-cell transplantation is the only existing cure but is limited to specialised clinical centres and remains inaccessible for most patients. Induction of fetal haemoglobin production is a promising strategy for the treatment of sickle-cell disease. In this Series paper, we review scientific breakthroughs in epidemiology, genetics, and molecular biology that have brought reactivation of fetal haemoglobin to the forefront of sickle-cell disease research. Improved knowledge of the regulation of fetal haemoglobin production in human beings and the development of genome editing technology now support the design of innovative therapies for sickle-cell disease that are based on fetal haemoglobin.
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