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Genetic Engineering and Manufacturing of Hematopoietic Stem Cells

18 Mar 2017

Abstract excerpt

The marketing approval of genetically engineered hematopoietic stem cells (HSCs) as the first-line therapy for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) is a tribute to the substantial progress that has been made regarding HSC engineering in the past decade. Reproducible manufacturing of high-quality, clinical-grade, genetically engineered HSCs is the...

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PMID: 28480310
DOI: 10.1016/j.omtm.2017.03.003

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