Abstract
Treatment of genetic deficiency diseases, the so-called inborn errors of metabolism has been fairly limited in scope. Whereas there has been significant success with injections of the missing gene product in cases such as diabetes and hemophilia, management of most genetic diseases has been limited to treatment of various clinical symptoms and not the underlying disease state. For the past decade, many groups have been working toward the goal of gene therapy, i.e., the correction of genetic deficiency diseases by the introduction of a correct copy of the defective gene into affected individuals. Many different diseases have been targeted (see ref. 1 for review) utilizing a variety of cells and animal models (1–14). Although many groups have been able to obtain high levels of gene expression in vitro, in vivo expression has been disappointing. In most cases, while the gene product could be detected within hours after introduction into the host, high level expression was not detectable after 30–45 d (4,5,11,12,14–19).
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Morgan, R. A. and Anderson, W. F. (1993) Human gene therapy. Ann. Rev. Biochem. 62, 191–217.
Lozier, J. N. and Brinkhous, K. M. (1994) Gene therapy and the hemophilias. JAMA 27, 47–51.
Anson, D. S., Hock, R. A., Austen, D., Smith, K. J., Brownlee, G. G., Verma, I. M., and Miller, A. D. (1987) Towards gene therapy for hemophilia B. Mol. Biol. Med. 4, 11–20.
Zhou, J. M., Dai, Y. F., Qiu, X. F., Hou, G. Y., Akira, Y., and Xue, J. L. (1993) Expression of human factor IX cDNA in mice by implants of genetically modified skin fibroblasts from a hemophilia B patient. Sci. China (B) 36, 1082–1092.
Kay, M. A., Landen, C. N., Rothenberg, S. R., Taylor, L. A., Leland, F., Wiehle, S., Fang, B., Bellinger, D., Finegold, M., Thompson, A. R., Read, M. S., Brinkhous, K. M., and Woo, S. L. C. (1994) In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc. Natl. Acad. Sci. USA 91, 2353–2357.
Li, Q., Kay, M. A., Finegold, M., Stratford-Perricaudet, L. D., and Woo, S. L. (1993) Assessment of recombinant adenoviral vectors for hepatic gene therapy. Hum. Gene. Ther. 4, 403–409.
Liu, H.-W., Ofosu, F. A., and Chang, P. L. (1993) Expression of human factor IX by microencapsulated recombinant fibroblasts. Hum. Gene. Ther. 4, 291–301.
Yao, S.-N., Wilson, J. M., Nabel, E. G., Kurachi, S., Hachiya, H. L., and Kurachi, K. (1991) Expression of human factor IX in rat capillary endothelial cells toward somatic gene therapy for hemophilia B. Proc. Natl. Acad. Sci. USA 88, 8101–8105.
Anson, D. S., Austen, D. E. G., and Brownlee, G. G. (1985) Expression of active human clotting factor IX from recombinant DNA clones in mammalian cells. Nature 315, 683–685.
de la Salle, H., Altenburger, W., Elkaim, R., Dott, K., Dieterle, A., Drillien, R., Cazenave, J.-P., Tolstoshev, P., and Lecocq, J.-P. (1985) Active gamma-carboxylated human factor IX expressed using recombinant DNA techniques. Nature 316, 268–270.
Axelrod, J. H., Read, M. S., Brinkhous, K. M., and Verma, I. M. (1990) Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophiliac dogs. Proc. Natl._Acad. Sci. USA 87, 5173–5177.
Palmer, T. D., Thompson, A. R., and Miller, D. (1989) Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B. Blood 73, 438–445.
Armentano, D., Thompson, A. R., Darlington, G., and Woo, S. L. C. (1990) Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer. Potential for gene therapy of hemophilia B. Proc. Natl. Acad. Sci. USA 87, 6141–6145.
Palmer, T. D., Rosman, G. J., Osborne, W. R., and Miller, A. D. (1991) Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc. Natl._Acad. Sci. USA 88, 1330–1334.
Yao, S.-N. and Kurachi, K. (1992) Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. USA. 89, 3357–3361.
St. Louis, D. and Verma, I. M. (1988) An alternative approach to somatic cell gene therapy. Proc. Natl. Acad. Sci. USA 85, 3150–3154.
Scharfmann, R., Axelrod, J. H., and Verma, I. M. (1991) Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc. Natl._Acad. Sci. USA 88, 4626–4630.
Dai, Y., Roman, M., Naviaux, R. K., and Verma, I. M. (1992) Gene therapy via primary myoblasts. long-term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89, 10,892–10,895.
Miyanohara, A., Johnson, P. A., Elam, R. L., Dai, Y., Witztum, J. L., Verma, I. M., and Friedmann, T. (1992) Direct gene transfer to the liver with herpes simplex virus type 1 vectors. transient production of physiologically relevant levels of circulating factor IX. New Biol. 4, 238–246.
Thompson, J. A., Haudenschild, C. C., Anderson, K. D., DiPietro, J. M., Anderson, W. F., and Maclag, T. (1989) Heparin-binding growth factor 1 induces the formation of organoid neovascular structures in vivo. Proc. Natl._Acad. Sci. USA 86, 7928–7932.
Brauker, J. H., Carr-Brendel, V., Martinson, L. A., Crudele, J., Johnston, W. D., and Johnson, R. C. (1995) Neovascularization of synthetic membranes directed by membrane architecture. J. Biomed. Mat. Res. 29, 1517–1524.
Brauker, J., Martinson, L. A., Young, S. K., and Johnson, R. C. (1996) Local inflammatory response around diffusion chambers containing xenografts. Transplantation 61, 1671–1677.
Algire, G. H., Weaver, J. M., and Prehn, R. T. (1954) Growth of cells in vivo in diffusion chambers I. survival of homografts in immunized mice. J. Natl. Cancer Inst. 15, 493–507.
Larson, P. J. and High, K. A. (1992) Biology of inherited coagulopathies factor IX Hematol. Oncol. Clin. North. Am. 6, 999–10
Kurachi, K., Furukawa, M., Yao, S. N., and Kurachi, S. (1992) Biology of factor IX. Hematol. Oncol. Clin. North Am. 6, 991–997.
Carr-Brendel, V., Lazier, J., Thomas, T., Saeed, B., Young, S., Crudele, J., Martinson, L., Roche, B., Boggs, D., Pauley, R., Maryanov, D., Josephs, S., High, K., Johnson, R., and Brauker, J. (1993) An immunoisolation device for implantation of genetically engineered cells: long term expression of factor IX in rats. J. Cell Biochem. 17E, 224.
Delves, M. N. (1994) Cellular Immunology Labfax 1994 Academic, New York.
Author information
Authors and Affiliations
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 1997 Humana Press Inc., Totowa, NJ
About this protocol
Cite this protocol
Carr-Brendel, V.E. et al. (1997). Transplantation of Cells in an immunoisolation Device for Gene Therapy. In: Tuan, R.S. (eds) Recombinant Protein Protocols. Methods in Molecular Biology™, vol 63. Humana Press. https://doi.org/10.1385/0-89603-481-X:373
Download citation
DOI: https://doi.org/10.1385/0-89603-481-X:373
Publisher Name: Humana Press
Print ISBN: 978-0-89603-481-5
Online ISBN: 978-1-59259-549-5
eBook Packages: Springer Protocols