By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Biotechnology have been presented. According to news originating from Seattle, Washington, by NewsRx correspondents, research stated, "A major hurdle for hematopoietic stem cell (HSC) gene therapy for inherited bone marrow disorders, including Fanconi anemia (FA), is adequate engraftment of gene-modified cells. A phenotypic defect in DNA repair renders FA patients sensitive to alkylating agents such as cyclophosphamide (Cy); however, at lower doses, Cy is well tolerated in the FA transplant setting."
Our news journalists obtained a quote from the research from Fred Hutchinson Cancer Research Center, "We tested whether non-alkylating agents could replace Cy for pretransplant conditioning to enhance engraftment of FANCA gene-modified hematopoietic cells. We compared Cy preconditioning with fludarabine (Flu) or cytarabine (AraC) or no conditioning as a control in fanca (-/-) mutant mice receiving gene-modified bone marrow cells. Only mice conditioned with Cy exhibited appreciable engraftment of gene-modified cells by PCR and resistance to mitomycin C (MMC). Cy administration following transplantation increased gene marking levels in all animals treated, but highest gene marking and corresponding MMC resistance were observed in mice receiving Cy pre-and posttransplantation. Importantly, no cytogenetic abnormalities were observed in Cy-treated mice."
According to the news editors, the research concluded: "Cy is an effective and superior preparative regimen with respect to engraftment of lentivirus-transduced cells and functional correction in fanca (-/-) mice. Thus, appropriately dosed Cy may provide a suitable conditioning regimen for FA patients undergoing HSC gene therapy."
For more information on this research see: Cyclophosphamide promotes engraftment of gene-modified cells in a mouse model of Fanconi anemia without causing cytogenetic abnormalities. Journal of Molecular Medicine, 2012;90(11):1283-94. Journal of Molecular Medicine can be contacted at: Springer, 233 Spring Street, New York, NY 10013, USA. (Springer - www.springer.com; Journal of Molecular Medicine - www.springerlink.com/content/0946-2716/)
The news correspondents report that additional information may be obtained from J.E. Adair, Division of Clinical Research, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue North, Seattle, WA 98109-1024, United States. Additional authors for this research include X. Zhao, S. Chien, M. Fang, M.E. Wohlfahrt, G.D. Trobridge, J.A. Taylor, B.C. Beard, H.P. Kiem and P.S Becker (see also technology.html">Biotechnology).
The publisher's contact information for the Journal of Molecular Medicine is: Springer, 233 Spring Street, New York, NY 10013, USA.
Keywords for this news article include: Antineoplastics, Biotechnology, Pharmaceuticals, Drugs, Seattle, Washington, Hypoplastic, Gene Therapy, Hydrocarbons, United States, Bioengineering, Fanconi Anemia, Cyclophosphamide, Alkylating Agents, Congenital Anemia, Mustard Compounds, Metabolic Diseases, Bone Marrow Diseases, Hematologic Diseases.
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