Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer
Joint Authors
Habi, Ouassila
Bourdages, Valérie
Girard, Johanne
Carreau, Madeleine
Delisle, Marie-Chantal
Source
Issue
Vol. 2010, Issue 2010 (31 Dec. 2010), pp.1-13, 13 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2010-03-16
Country of Publication
Egypt
No. of Pages
13
Main Subjects
Abstract EN
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.
American Psychological Association (APA)
Habi, Ouassila& Girard, Johanne& Bourdages, Valérie& Delisle, Marie-Chantal& Carreau, Madeleine. 2010. Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer. Anemia،Vol. 2010, no. 2010, pp.1-13.
https://search.emarefa.net/detail/BIM-510501
Modern Language Association (MLA)
Habi, Ouassila…[et al.]. Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer. Anemia No. 2010 (2010), pp.1-13.
https://search.emarefa.net/detail/BIM-510501
American Medical Association (AMA)
Habi, Ouassila& Girard, Johanne& Bourdages, Valérie& Delisle, Marie-Chantal& Carreau, Madeleine. Correction of Fanconi Anemia Group C Hematopoietic Stem Cells Following Intrafemoral Gene Transfer. Anemia. 2010. Vol. 2010, no. 2010, pp.1-13.
https://search.emarefa.net/detail/BIM-510501
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-510501