Functional correction of fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector

Molecular Therapy : the Journal of the American Society of Gene Therapy
K YamadaC E Walsh

Abstract

Lentiviral vectors transduce nondividing hematopoietic cells more efficiently than other currently available vector systems. Here we report the results of human hematopoietic cell gene transfer using lentiviral vectors based upon human immunodeficiency virus (HIV-1) and equine infectious anemia virus (EIAV). EIAV is a nonprimate lentivirus and is severely restricted in its host range to horses and closely related equines. We employed the EIAV vector system to test for gene transfer to human Fanconi anemia (FA) hematopoietic cells by comparison with HIV-1- and Moloney murine leukemia virus-based systems. Fanconi anemia is characterized by bone marrow failure secondary to stem cell dysfunction. Fanconi anemia group C EBV-transformed lymphoblasts were transduced with all three viral vectors. Phenotypic correction of FA cells, as measured by mitomycin C drug resistance, was observed with a similar efficiency in all vector systems. This is the first description of lentiviral correction of FA cells and suggests that lentiviral vectors may be useful for FA gene transfer.

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Citations

Feb 8, 2003·DNA and Cell Biology·Ricardo Quinonez, Richard E Sutton
Dec 6, 2007·BMC Biochemistry·Céline Jacquemont, Toshiyasu Taniguchi
Sep 2, 2010·Future Neurology·Robert A MarrDaniel A Peterson
Feb 8, 2006·Proceedings of the National Academy of Sciences of the United States of America·Odile Cohen-HaguenauerMichel Marty
Aug 16, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Elena K SiapatiDominique Bonnet
Apr 30, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·J P O'RourkeB A Bunnell
Oct 8, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·Kaoru YamadaChristopher E Walsh
May 10, 2002·BioEssays : News and Reviews in Molecular, Cellular and Developmental Biology·Shamim I AhmadSandra H Kirk
Mar 20, 2004·Expert Review of Cardiovascular Therapy·Roland W Herzog, Volder R Arruda
Nov 22, 2005·The Journal of Gene Medicine·K S BalagganR R Ali

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