New Gene Therapy Findings Reported from University of Zurich (Human miR223 Promoter as a Novel Myelo-Specific Promoter for Chronic Granulomatous Disease Gene Therapy)
By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Biotechnology have been presented. According to news originating from Zurich, Switzerland, by NewsRx correspondents, research stated, "Targeting transgene expression to specific hematopoietic cell lineages could contribute to the safety of retroviral vectors in gene therapeutic applications. Chronic granulomatous disease (CGD), a defect of phagocytic cells, can be managed by gene therapy, using retroviral vectors with targeted expression to myeloid cells."
Our news journalists obtained a quote from the research from the University of Zurich, "In this context, we analyzed the myelospecificity of the human miR223 promoter, which is known to be strongly upregulated during myeloid differentiation, to drive myeloid-restricted expression of p47(phox) and gp91(phox) in mouse models of CGD and in primary patient-derived cells. The miR223 promoter restricted the expression of p47(phox), gp91(phox), and green fluorescent protein (GFP) within self-inactivating (SIN) gamma-and lentiviral vectors to granulocytes and macrophages, with only marginal expression in lymphocytes or hematopoietic stem and progenitor cells. Furthermore, gene transfer into primary CD34+ cells derived from a p47(phox) patient followed by ex vivo differentiation to neutrophils resulted in restoration of Escherichia coli killing activity by miR223 promoter-mediated p47(phox) expression."
According to the news editors, the research concluded: "These results indicate that the miR223 promoter as an internal promoter within SIN gene therapy vectors is able to efficiently correct the CGD phenotype with negligible activity in hematopoietic progenitors, thereby limiting the risk of insertional oncogenesis and development of clonal dominance."
For more information on this research see: Human miR223 Promoter as a Novel Myelo-Specific Promoter for Chronic Granulomatous Disease Gene Therapy. Human Gene Therapy Methods, 2013;24(3):151-159. Human Gene Therapy Methods can be contacted at: Mary Ann Liebert, Inc, 140 Huguenot Street, 3RD Fl, New Rochelle, NY 10801, USA (see also Biotechnology).
The news correspondents report that additional information may be obtained from C. Brendel, University of Zurich, Cent Biol Lab, CH-8091 Zurich, Switzerland. Additional authors for this research include W. Hanseler, V. Wohlgensinger, M. Bianchi, S. Tokmak, L. Chen-Wichmann, E. Kuzmenko, N. Cesarovic, F. Nicholls, J. Reichenbach, R. Seger, M. Grez and U. Siler.
Keywords for this news article include: Biotechnology, Zurich, Europe, Hematology, Switzerland, Gene Therapy, Hematopoietic, Bioengineering, Chronic Granulomatous Disease
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