Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors
Author:
Publisher
Springer Science and Business Media LLC
Subject
Hematology
Link
http://link.springer.com/content/pdf/10.1186/s12959-016-0105-1.pdf
Reference47 articles.
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2. Roth DA, Tawa Jr NE, O’Brien JM, Treco DA, Selden RF. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med. 2001;344:1735–42.
3. Powell JS, Ragni MV, White 2nd GC, et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood. 2003;102:2038–45.
4. White 2nd GC. Gene therapy in hemophilia: clinical trials update. Thromb Haemost. 2001;86:172–7.
5. Doering CB, Gangadharan B, Dukart HZ, Spencer HT. Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity. Mol Ther. 2007;15:1093–9.
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2. CD14+/CD31+ monocytes expanded by UM171 correct hemophilia A in zebrafish upon lentiviral gene transfer of factor VIII;Blood Advances;2023-03-01
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5. Transduction of modified factor VIII gene improves lentiviral gene therapy efficacy for hemophilia A;Journal of Biological Chemistry;2021-12
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