Research on fanconi anemia reported by A. Jacome et al
2009 AUG 3 - (NewsRx.com) -- According to recent research from Madrid, Spain, "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," wrote A. Jacome and colleagues. The researchers concluded: "We propose a new approach for the gene therapy of FA based on the rapid transduction of unselected hematopoietic grafts with lentiviral vectors (LVs)." Jacome and colleagues published their study in Molecular Therapy (Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients. Molecular Therapy, 2009;17(6):1083-1092). For additional information, contact J.A. Bueren, CIEMAT, Division Hematopoyesis & Terapia Genica, Avenue Complutense 22, E-28040 Madrid, Spain. Publisher contact information for the journal Molecular Therapy is: Nature Publishing Group, 75 Varick St., 9TH Flr, New York, NY 10013-1917, USA. Keywords: Spain, Madrid, Adenosine, Adenosine Deaminase, Biotechnology, Bone Marrow, Chronic Granulomatous Disease, Clinical Trial Research, Deaminase, Dermatology, Drugs, Enzyme Research, Enzymes, Enzymology, Fanconi Anemia, Gene Therapy, Genetics, Hematology, Hematopoietic, Immunology, Molecular Research, Molecular Therapies, Pharmaceuticals, Progenitor Cell, Proteins, Proteomics, Severe Combined Immunodeficiency, Stem Cell Research, Treatment. This article was prepared by Biotech Business Week editors from staff and other reports. Copyright 2009, Biotech Business Week via NewsRx.com.
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