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Journal article
Engineering Factor Viii for Hemophilia Gene Therapy
Journal of genetic syndrome & gene therapy, v 1, S1
21 Dec 2011
PMID: 23565342
Abstract
Current treatment of hemophilia A by intravenous infusion of factor VIII (fVIII) concentrates is very costly and has a potential adverse effect of developing inhibitors. Gene therapy, on the other hand, can potentially overcome these limitations associated with fVIII replacement therapy. Although hemophilia B gene therapy has achieved promising outcomes in human clinical trials, hemophilia A gene therapy lags far behind. Compared to factor IX, fVIII is a large protein which is difficult to express at sustaining therapeutic levels when delivered by either viral or non-viral vectors. To improve fVIII gene delivery, numerous strategies have been exploited to engineer the fVIII molecule and overcome the hurdles preventing long term and high level expression. Here we reviewed these strategies, and discussed their pros and cons in human gene therapy of hemophilia A.
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Details
- Title
- Engineering Factor Viii for Hemophilia Gene Therapy
- Creators
- Sean A. Roberts - Sol Sherry Thrombosis Research Center, Philadelphia PA 19140, USA Department of Microbiology and Immunology, Temple University Medical School, Philadelphia PA 19140, USA Department of Biology, Drexel University, Philadelphia, PA 19104, USABiao Dong - Sol Sherry Thrombosis Research Center, Philadelphia PA 19140, USA Department of Microbiology and Immunology, Temple University Medical School, Philadelphia PA 19140, USA Department of Biology, Drexel University, Philadelphia, PA 19104, USAJenni A. Firrman - Sol Sherry Thrombosis Research Center, Philadelphia PA 19140, USA Department of Microbiology and Immunology, Temple University Medical School, Philadelphia PA 19140, USA Department of Biology, Drexel University, Philadelphia, PA 19104, USAAndrea R. Moore - Sol Sherry Thrombosis Research Center, Philadelphia PA 19140, USA Department of Microbiology and Immunology, Temple University Medical School, Philadelphia PA 19140, USA Department of Biology, Drexel University, Philadelphia, PA 19104, USANianli Sang - Drexel UniversityWeidong Xiao - Sol Sherry Thrombosis Research Center, Philadelphia PA 19140, USA Department of Microbiology and Immunology, Temple University Medical School, Philadelphia PA 19140, USA Department of Biology, Drexel University, Philadelphia, PA 19104, USA
- Publication Details
- Journal of genetic syndrome & gene therapy, v 1, S1
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Biology
- Other Identifier
- 991021211720204721