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Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.

Abstract：

: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 foundation for broadening the application of this technology. Herein, the current state-of-the-art manufacturing platforms to genetically engineer HSCs as well as the challenges pertaining to production standardization and product characterization are addressed in the context of primary immunodeficiency diseases (PIDs) and other monogenic disorders.

journal_name

Mol Ther Methods Clin Dev

journal_title

Molecular therapy. Methods & clinical development

authors

Wang X,Rivière I

doi

10.1016/j.omtm.2017.03.003

subject

Has Abstract

pub_date

2017-03-18 00:00:00

pages

96-105

issn

2329-0501

pii

S2329-0501(17)30048-7

journal_volume

5

pub_type

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