Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2010-10

AUTHORS

Pamela S. Becker, Jason A. Taylor, Grant D. Trobridge, Xin Zhao, Brian C Beard, Sylvia Chien, Jennifer Adair, Donald B. Kohn, John E. Wagner, Akiko Shimamura, Hans-Peter Kiem

ABSTRACT

One of the major hurdles for the development of gene therapy for Fanconi anemia (FA) is the increased sensitivity of FA stem cells to free radical-induced DNA damage during ex vivo culture and manipulation. To minimize this damage, we have developed a brief transduction procedure for lentivirus vector-mediated transduction of hematopoietic progenitor cells from patients with Fanconi anemia complementation group A (FANCA). The lentiviral vector FancA-sW contains the phosphoglycerate kinase promoter, the FANCA cDNA, and a synthetic, safety-modified woodchuck post transcriptional regulatory element (sW). Bone marrow mononuclear cells or purified CD34(+) cells from patients with FANCA were transduced in an overnight culture on recombinant fibronectin peptide CH-296, in low (5%) oxygen, with the reducing agent, N-acetyl-L-cysteine (NAC), and a combination of growth factors, granulocyte colony-stimulating factor (G-CSF), Flt3 ligand, stem cell factor, and thrombopoietin. Transduced cells plated in methylcellulose in hypoxia with NAC showed increased colony formation compared with 21% oxygen without NAC (P<0.03), showed increased resistance to mitomycin C compared with green fluorescent protein (GFP) vector-transduced controls (P<0.007), and increased survival. Thus, combining short transduction and reducing oxidative stress may enhance the viability and engraftment of gene-corrected cells in patients with FANCA. More... »

PAGES

1244

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/gt.2010.62

DOI

http://dx.doi.org/10.1038/gt.2010.62

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1049963999

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/20485382


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