News Column

Researchers' Work from Fujian Medical University Focuses on Gene Therapy

February 20, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Research findings on Biotechnology are discussed in a new report. According to news reporting originating from Fujian, People's Republic of China, by NewsRx correspondents, research stated, "Our previous studies have demonstrated that platelet FVIII (2bF8) gene therapy can improve hemostasis in hemophilia A mice, even in the presence of inhibitory antibodies, but none of our studies has targeted human cells. Here, we evaluated the feasibility for lentivirus (LV)-mediated human platelet gene therapy of hemophilia A. Human platelet FVIII expression was introduced by 2bF8LV-mediated transduction of human cord blood (hCB) CD34(+) cells followed by xenotransplantation into immunocompromised NSG mice or NSG mice in an FVIIInull background (NSGF8KO)."

Our news editors obtained a quote from the research from Fujian Medical University, "Platelet FVIII was detected in all recipients that received 2bF8LV-transduced hCB cells as long as human platelet chimerism persisted. All NSGF8KO recipients (n = 7) that received 2bF8LV-transduced hCB cells survived tail clipping if animals had greater than 2% of platelets derived from 2bF8LV-transduced hCB cells, whereas 5 of 7 survived when human platelets were 0.3% to 2%. Whole blood clotting time analysis confirmed that hemostasis was improved in NSGF8KO mice that received 2bF8LV-transduced hCB cells."

According to the news editors, the research concluded: "We demonstrate, for the first time, the feasibility of 2bF8LV gene delivery to human hematopoietic stem cells to introduce FVIII expression in human platelets and that human platelet-derived FVIII can improve hemostasis in hemophilia A."

For more information on this research see: Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells. Blood, 2014;123(3):395-403. Blood can be contacted at: Amer Soc Hematology, 2021 L St NW, Suite 900, Washington, DC 20036, USA. (American Society of Hematology -; Blood -

The news editors report that additional information may be obtained by contacting Q.Z. Shi, Fujian Med Univ, Union Hosp, Fujian Inst Hematol, Fujian Prov Key Lab Hematol, Fuzhou, Fujian, People's Republic of China. Additional authors for this research include E.L. Kuether, Y.Y. Chen, J.A. Schroeder, S.A. Fahs and R.R. Montgomery (see also Biotechnology).

Keywords for this news article include: Asia, Biotechnology, Fujian, Cord Blood, Gene Therapy, Hemophilia A, Bioengineering, Blood Transfusion, Stem Cell Research, Hematologic Diseases, Transfusion Medicine, Hemorrhagic Disorders, People's Republic of China, Hemic and Lymphatic Diseases, Coagulation Protein Disorders, Inherited Blood Coagulation Disorders

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Source: Gene Therapy Weekly

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