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Engraftment of Hematopoietic Progenitor Cells Transduced with the Fanconi Anemia Group C Gene (FANCC)
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“The success of gene therapy approaches to FA in a clinical setting has been limited by a combination of factors, including the paucity of autologous CD34+ cells available as targets for gene correction, reservations about the use of cytoreductive therapy to “make space” for the autologous graft, and potentially defective engraftment of FA HSC following prolonged in vitro processing[33–35].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [34]
In the citing paper
“While vector sequences were detectable transiently in peripheral blood and bone marrow mononuclear cells of several patients in both trials, neither trial achieved prolonged engraftment of gene modified HSC[34,35].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [34]
In the citing paper
“In addition, the predicted selective advantage of genetically corrected HSC over FANC-deficient HSC has not been observed in either trial.”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [34]
In the citing paper
“Despite considerable challenges related to the procurement of autologous CD34+ HSC/P, two clinical gene therapy trials involving a total of 7 patients with FANCA and FANCC mutations have been conducted and these studies established the feasibility of gammaretroviral gene transfer of FA genes into human hematopoietic progenitor cells[34,35].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [34]
In the citing paper
“In both previous FA gene therapy trials, in vitro culture of FA HSCs lasted up to 84 hours[34,35].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [34]
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Cited by
The Fanconi anaemia/BRCA pathway
D'Andrea, A. D., & Grompe, M. (2003) · Nature Reviews Cancer · doi:10.1038/nrc970 · Cited by 822
Stem Cell Collection and Gene Transfer in Fanconi Anemia
Kelly, P. F., Radtke, S., Kalle, C. V., Balcik, B., Bohn, K., Mueller, R., Schuesler, T., Haren, M., Reeves, L., Cancelas, J. A., et al. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300033 · Cited by 180
Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia
Müller, L. U. W., & Williams, D. A. (2009) · Mutation Research - Fundamental and Molecular Mechanisms of Mutagenesis · doi:10.1016/j.mrfmmm.2009.03.010 · Cited by 31
Metadata
- Authors
- Liu, J. M., Kim, S., Read, E. J., Futaki, M., Dokal, I., Carter, C. S., Leitman, S. F., Pensiero, M., Young, N. S., & Walsh, C. E.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 1999-09-20
- Volume
- 10
- Issue
- 14
- Pages
- 2337-2346
- ISSN
- 1043-0342
- 1557-7422
- Cited by
- 117
- References
- 0
- Language
- en
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- CoChat. (2026). Citation check: Engraftment of Hematopoietic Progenitor Cells Transduced with the Fanconi Anemia Group C Gene (FANCC). https://app.cochat.ai/papers/10.1089/10430349950016988
- The paper
- Liu, J. M., Kim, S., Read, E. J., Futaki, M., Dokal, I., Carter, C. S., Leitman, S. F., Pensiero, M., Young, N. S., & Walsh, C. E. (1999). Engraftment of Hematopoietic Progenitor Cells Transduced with the Fanconi Anemia Group C Gene (FANCC). Human Gene Therapy. https://doi.org/10.1089/10430349950016988