Genetic engineering and manufacturing of hematopoietic stem cells Journal Article

Authors: Wang, X.; Rivière, I.
Article Title: 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.
Keywords: severe combined immunodeficiency; phase i/ii trial; cells; chronic granulomatous-disease; bone-marrow; lentiviral vector; retroviral vectors; mobilized peripheral-blood; wiskott-aldrich syndrome; adenosine-deaminase deficiency; cd34(+)
Journal Title: Molecular Therapy - Methods & Clinical Development
Volume: 5
ISSN: 2329-0501
Publisher: Nature Publishing Group  
Date Published: 2017-06-16
Start Page: 96
End Page: 105
Language: English
ACCESSION: WOS:000406299600012
DOI: 10.1016/j.omtm.2017.03.003
PMCID: PMC5415326
PUBMED: 28480310
Notes: Review -- Source: Wos
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MSK Authors
  1. Isabelle C Riviere
    169 Riviere
  2. Xiuyan Wang
    70 Wang