ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03157804 (Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A), which is not on this map. Cited by 21 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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.
Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)
Who cites it
21 citing papers in PubMed, 48 citations in OpenAlex.
- Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A.Haematologica · 2018Trial
- Article
- Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo.Gene therapy · 2023Article
- Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes.Molecular therapy. Methods & clinical development · 2021Article
- TALEN mediated gene editing in a mouse model of Fanconi anemia.Scientific reports · 2020Article
- Therapeutic gene editing in CD34EMBO molecular medicine · 2017Article
- Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent.Blood advances · 2017Article
- Biological and functional characterization of bone marrow-derived mesenchymal stromal cells from patients affected by primary immunodeficiency.Scientific reports · 2017Article
- The PI3K/Akt1 pathway enhances steady-state levels of FANCL.Molecular biology of the cell · 2013Article
- Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells.Molecular therapy. Nucleic acids · 2013Article
- Lentiviral vectors and cardiovascular diseases: a genetic tool for manipulating cardiomyocyte differentiation and function.Gene therapy · 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
- Ex vivo gene transfer and correction for cell-based therapies.Nature reviews. Genetics · 2011Review
- High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy.Human gene therapy · 2010Article
- Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.Gene therapy · 2010Article
- Lentiviral vectors in gene therapy: their current status and future potential.Archivum immunologiae et therapiae experimentalis · 2010Review
- A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells.Nature protocols · 2010Article
- Genetic correction of hematopoiesis in Fanconi anemia: the case for a non-HSC-autonomous defect.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.Nature · 2009Article
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Authors and funding
17 authors at 6 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Previous clinical trials based on the genetic correction of purified CD34(+) cells with gamma-retroviral vectors have demonstrated clinical efficacy in different monogenic diseases, including X-linked severe combined immunodeficiency, adenosine deaminase deficient severe combined immunodeficiency and chronic granulomatous disease. Similar protocols, however, failed to engraft Fanconi anemia (FA) patients with genetically corrected cells. In this study, we first aimed to correlate the hematological status of 27 FA patients with CD34(+) cell values determined in their bone marrow (BM). Strikingly, no correlation between these parameters was observed, although good correlations were obtained when numbers of colony-forming cells (CFCs) were considered. Based on these results, and because purified FA CD34(+) cells might have suboptimal repopulating properties, we investigated the possibility of genetically correcting unselected BM samples from FA patients. Our data show that the lentiviral transduction of unselected FA BM cells mediates an efficient phenotypic correction of hematopoietic progenitor cells and also of CD34(-) mesenchymal stromal cells (MSCs), with a reported role in hematopoietic engraftment. Our results suggest that gene therapy protocols appropriate for the treatment of different monogenic diseases may not be adequate for stem cell diseases like FA. We propose a new approach for the gene therapy of FA based on the rapid transduction of unselected hematopoietic grafts with lentiviral vectors (LVs).
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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.