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Stem Cell Collection and Gene Transfer in Fanconi Anemia
Open accessAbstract
Fanconi anemia (FA) is a rare genetic syndrome characterized by progressive bone marrow failure (BMF), congenital anomalies, and a predisposition to malignancy. Successful gene transfer into hematopoietic stem cells (HSCs) could reverse BMF in this disease. We developed clinical trials to determine whether a sufficient number of CD34(+) stem cells could be collected for gene modification and to evaluate the safety and efficacy of HSC-corrective gene transfer in FA genotype A (FANCA) patients. Here, we report that FA patients have significant depletion of their BM CD34(+) cell compartment even before severe pancytopenia is present. However, oncoretroviral-mediated ex vivo gene transfer was efficient in clinical scale in FA-A cells, leading to reversal of the cellular phenotype in a significant percentage of CD34(+) cells. Re-infusion of gene-corrected products in two patients was safe and well tolerated and accompanied by transient improvements in hemoglobin and platelet counts. Gene correction was transient, likely owing to the low dose of gene-corrected cells infused. Our early experience shows that stem cell collection is well tolerated in FA patients and suggests that collection be considered as early as possible in patients who are potential candidates for future gene transfer trials.
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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 [35]
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 [35]
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 [35]
In the citing paper
“However, broad inter-individual variability of efficacy exists in normal healthy subjects, patients pre-treated with chemotherapy, and patients with Fanconi anemia[34,35,69,81].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [35]
In the citing paper
“Thus, the target CD34+ cell dose of 2×106 per kg future weight was not obtained in any patient.”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [35]
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Citations
Cites
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A 20-year perspective on the International Fanconi Anemia Registry (IFAR)
Kutler, D. I., Singh, B., Satagopan, J., Batish, S. D., Berwick, M., Giampietro, P. F., Hanenberg, H., & Auerbach, A. D. (2003) · Blood · doi:10.1182/blood-2002-07-2170 · Cited by 618
Self-citationThe Fanconi Anemia/BRCA pathway: new faces in the crowdPreprint
Kennedy, R. D., & D’Andrea, A. D. (2005) · Genes & Development · doi:10.1101/gad.1370505 · Cited by 474
Modulation of hematopoietic stem cell homing and engraftment by CD26
Christopherson, K. W., Hangoc, G., Mantel, C. R., & Broxmeyer, H. E. (2004) · Science · doi:10.1126/science.1097071 · Cited by 452
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Metadata
- Authors
- Kelly, P. F., Radtke, S., Kalle, C. V., Balcik, B., Bohn, K., Mueller, R., Schuesler, T., Haren, M., Reeves, L., Cancelas, J. A., Leemhuis, T., Harris, R., Auerbach, A. D., Smith, F. O., Davies, S. M., & Williams, D. A.
- Affiliations
- Cincinnati Children's Hospital Medical Center (US)
- University of Cincinnati (US)
- Rockefeller University (US)
- Corresponding author
- Williams, David A
- ORCID
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2007-01
- Volume
- 15
- Issue
- 1
- Pages
- 211-219
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2007940575
- Cited by
- 180
- References
- 46
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 7 · 2023: 8 · 2022: 3 · 2021: 8 · 2020: 8 · 2019: 5 · 2018: 8 · 2017: 9 · 2016: 13 · 2015: 9 · 2014: 6 · 2013: 15 · 2012: 16
- FWCI
- 6.45
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health
- National Heart, Lung, and Blood Institute (R37HL032987, R37 HL23987, R01HL081499)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
- Health Sciences › Medicine › Hematology › Blood disorders and treatments
- Keywords
- Fanconi anemia, FANCA, hematopoietic stem cells, CD34+ cell collection, ex vivo gene transfer, oncoretroviral vectors, bone marrow failure, stem cell depletion, clinical trial safety and efficacy
- MeSH terms
- Adolescent, Bone Marrow, Cell Separation, Child, Child, Preschool, Fanconi Anemia, Humans, Infant, Stem Cells, Genetic Therapy, DNA, Complementary, Antigens, CD34, Transgenes, Fanconi Anemia Complementation Group A Protein
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- CoChat. (2026). Citation check: Stem Cell Collection and Gene Transfer in Fanconi Anemia. https://app.cochat.ai/papers/10.1038/sj.mt.6300033
- The paper
- Kelly, P. F., Radtke, S., Kalle, C. V., Balcik, B., Bohn, K., Mueller, R., Schuesler, T., Haren, M., Reeves, L., Cancelas, J. A., Leemhuis, T., Harris, R., Auerbach, A. D., Smith, F. O., Davies, S. M., & Williams, D. A. (2007). Stem Cell Collection and Gene Transfer in Fanconi Anemia. Molecular Therapy. https://doi.org/10.1038/sj.mt.6300033
