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Habi O, Girard J, Bourdages V, et al. Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer. Anemia. 2010;2010doi: 10.1155/2010/947816.
Habi, O., Girard, J., Bourdages, V., Delisle, M. C., & Carreau, M. (2010). Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer. Anemia, 2010. https://doi.org/10.1155/2010/947816
Habi, Ouassila, et al. "Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer." Anemia vol. 2010 (2010). doi: https://doi.org/10.1155/2010/947816
Habi O, Girard J, Bourdages V, Delisle MC, Carreau M. Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer. Anemia. 2010;2010. doi: 10.1155/2010/947816. PMID: 20885913; PMCID: PMC2946959.
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Anemia. 2010;2010. doi: 10.1155/2010/947816.

Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer.

Anemia

Ouassila Habi, Johanne Girard, Valérie Bourdages, Marie-Chantal Delisle, Madeleine Carreau

Affiliations

  1. Unité de Recherche en Pédiatrie, Department of Pediatrics, Université Laval, CHUQ-CHUL, RC-9800, 2705 Boulevard Laurier, Québec, QC, Canada G1V 4G2.

PMID: 20885913 PMCID: PMC2946959 DOI: 10.1155/2010/947816

Abstract

The main cause of morbidity and mortality in Fanconi anemia patients is the development of bone marrow (BM) failure; thus correction of hematopoietic stem cells (HSCs) through gene transfer approaches would benefit FA patients. However, gene therapy trials for FA patients using ex vivo transduction protocols have failed to provide long-term correction. In addition, ex vivo cultures have been found to be hazardous for FA cells. To circumvent negative effects of ex vivo culture in FA stem cells, we tested the corrective ability of direct injection of recombinant lentiviral particles encoding FancC-EGFP into femurs of FancC(-/-) mice. Using this approach, we show that FancC(-/-) HSCs were efficiently corrected. Intrafemoral gene transfer of the FancC gene prevented the mitomycin C-induced BM failure. Moreover, we show that intrafemoral gene delivery into aplastic marrow restored the bone marrow cellularity and corrected the remaining HSCs. These results provide evidence that targeting FA-deficient HSCs directly in their environment enables efficient and long-term correction of BM defects in FA.

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