Research ArticleSICKLE CELL DISEASE

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

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Science Translational Medicine  12 Oct 2016:
Vol. 8, Issue 360, pp. 360ra134
DOI: 10.1126/scitranslmed.aaf9336

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Hammering out the sickle cell mutation

Sickle cell disease is a genetic disorder caused by a mutation in one of the hemoglobin genes, which causes deformation of red blood cells and results in occlusion of blood vessels, severe pain crises, and progressive organ injury. To correct the mutation that causes this disease, DeWitt et al. modified hematopoietic stem cells from sickle cell disease patients using a CRISPR/Cas9 gene editing approach. The authors showed that the corrected cells successfully engrafted in a mouse model and produced enough normal hemoglobin to have a potential clinical benefit in the setting of sickle cell disease.

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