Abstract
A number of different cell types including keratinocytes, hepatodytes, fibroblasts, myoblasts, and endothelial cells have been targeted for factor IX (RIX) gene replacement therapy.1–7 Following gene transfer, these cells have produced functional RIX because of their ability to gamma-carboxylate the appropriate “gla” domains and secrete the functional protein. Most efforts to date have employed an ex vivo gene transfer method in rodents and involve transfer of the F.IX gene into the cells via a recombinant retroviral vector and subsequent transplantation of the cells back into the animal. Two major problems encountered in these studies have been the rather low concentration of circulating F.IX and/or the loss of F.IX expression over time. The inability to achieve long-term expression is primarily the result of loss of the transduced cells or inactivation of the expression vectors.
Keywords
- Gene Transfer
- Partial Hepatectomy
- Inhibitor Formation
- Recombinant Adenoviral Vector
- Gene Transfer Method
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.
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© 1995 Plenum Press, New York
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Kay, M.A. (1995). Hepatic gene therapy for hemophilia B. In: Aledort, L.M., Hoyer, L.W., Lusher, J.M., Reisner, H.M., White, G.C. (eds) Inhibitors to Coagulation Factors. Advances in Experimental Medicine and Biology, vol 386. Springer, Boston, MA. https://doi.org/10.1007/978-1-4613-0331-2_22
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DOI: https://doi.org/10.1007/978-1-4613-0331-2_22
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