ArticleGene therapy2010
Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.
Article in Gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
20 citing papers in PubMed, 40 citations in OpenAlex.
- Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A.Haematologica · 2018Trial
- Lentiviral-mediated panErbB CAR-T cell therapy against head and neck squamous cell carcinomas for patients with Fanconi anemia.Molecular therapy. Oncology · 2025Article
- Simultaneous targeting of Tim3 and A2a receptors modulates MSLN-CAR T cell antitumor function in a human cervical tumor xenograft model.Frontiers in immunology · 2024Article
- The inhibitory receptors PD1, Tim3, and A2aR are highly expressed during mesoCAR T cell manufacturing in advanced human epithelial ovarian cancer.Cancer cell international · 2023Article
- TALEN mediated gene editing in a mouse model of Fanconi anemia.Scientific reports · 2020Article
- Highly Efficient Generation of Transgenically Augmented CAR NK Cells Overexpressing CXCR4.Frontiers in immunology · 2020Article
- MISTRG mice support engraftment and assessment of nonhuman primate hematopoietic stem and progenitor cells.Experimental hematology · 2019Article
- Efficient Enrichment of Gene-Modified Primary T Cells via CCR5-Targeted Integration of Mutant Dihydrofolate Reductase.Molecular therapy. Methods & clinical development · 2018Article
- Targeted mass spectrometry enables robust quantification of FANCD2 mono-ubiquitination in response to DNA damage.DNA repair · 2018Article
- Preclinical Optimization of a CD20-specific Chimeric Antigen Receptor Vector and Culture Conditions.Journal of immunotherapy (Hagerstown, Md. : 1997) · 2018Article
- Therapeutic gene editing in CD34EMBO molecular medicine · 2017Article
- Alcohol, stem cells and cancer.Genes & cancer · 2017Review
- Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy.Nature communications · 2016Article
- Preserved Activity of CD20-Specific Chimeric Antigen Receptor-Expressing T Cells in the Presence of Rituximab.Cancer immunology research · 2016Article
- Fanconi anemia and the cell cycle: new perspectives on aneuploidy.F1000prime reports · 2014Review
- Article
- Cyclophosphamide promotes engraftment of gene-modified cells in a mouse model of Fanconi anemia without causing cytogenetic abnormalities.Journal of molecular medicine (Berlin, Germany) · 2012Article
- Molecular pathogenesis and clinical management of Fanconi anemia.The Journal of clinical investigation · 2012Review
- Gene therapy for Fanconi anemia: one step closer to the clinic.Human gene therapy · 2012Article
- Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
Corrections and comments
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Authors and funding
11 authors at 5 institutions in 1 country.
Funding
Abstract
One of the major hurdles for the development of gene therapy for Fanconi anemia (FA) is the increased sensitivity of FA stem cells to free radical-induced DNA damage during ex vivo culture and manipulation. To minimize this damage, we have developed a brief transduction procedure for lentivirus vector-mediated transduction of hematopoietic progenitor cells from patients with Fanconi anemia complementation group A (FANCA). The lentiviral vector FancA-sW contains the phosphoglycerate kinase promoter, the FANCA cDNA, and a synthetic, safety-modified woodchuck post transcriptional regulatory element (sW). Bone marrow mononuclear cells or purified CD34(+) cells from patients with FANCA were transduced in an overnight culture on recombinant fibronectin peptide CH-296, in low (5%) oxygen, with the reducing agent, N-acetyl-L-cysteine (NAC), and a combination of growth factors, granulocyte colony-stimulating factor (G-CSF), Flt3 ligand, stem cell factor, and thrombopoietin. Transduced cells plated in methylcellulose in hypoxia with NAC showed increased colony formation compared with 21% oxygen without NAC (P<0.03), showed increased resistance to mitomycin C compared with green fluorescent protein (GFP) vector-transduced controls (P<0.007), and increased survival. Thus, combining short transduction and reducing oxidative stress may enhance the viability and engraftment of gene-corrected cells in patients with FANCA.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.