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Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles

3 Aug 2016

Abstract excerpt

Hematopoietic stem and progenitor cells (HSPCs) have great therapeutic potential because of their ability to both self-renew and differentiate. It has been proposed that, given their unique properties, a small number of genetically modified HSPCs could accomplish lifelong, corrective reconstitution of the entire hematopoietic system in patients with various hematologic disorders. Scientists have demonstrated that...

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PMID: 27483988
DOI: 10.1089/hum.2016.107

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