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December 9, 2013
07:05 EDTSGMOSangamo announces data from ZFP Therapeutic program
The data demonstrate that Sangamo's approach, using its proprietary and highly specific zinc finger nuclease gene-editing technology, enables permanent increase in the expression of fetal gamma-globin in adult red blood cells. This increase restores the normal balance of globin proteins that together form the oxygen-carrying hemoglobin of RBCs. In addition, the pre-clinical studies demonstrate that this can be accomplished at clinical-scale reproducibly achieving high levels of gene editing in hematopoietic stem cells.
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