Skip to main content
NCBI home page
Proceedings of the National Academy of Sciences of the United States of America logoLink to Proceedings of the National Academy of Sciences of the United States of America
. 1992 Feb 1;89(3):1138–1142. doi: 10.1073/pnas.89.3.1138

Long-term expression of human adenosine deaminase in vascular smooth muscle cells of rats: a model for gene therapy.

C M Lynch 1, M M Clowes 1, W R Osborne 1, A W Clowes 1, A D Miller 1
PMCID: PMC48401  PMID: 1736297

Abstract

Gene transfer into vascular smooth muscle cells in animals was examined by using recombinant retroviral vectors containing an Escherichia coli beta-galactosidase gene or a human adenosine deaminase (adenosine aminohydrolase, EC 3.5.4.4) gene. Direct gene transfer by infusion of virus into rat carotid arteries was not observed. However, gene transfer by infection of smooth muscle cells in culture and seeding of the transduced cells onto arteries that had been denuded of endothelial cells was successful. Potentially therapeutic levels of human adenosine deaminase activity were detected over 6 months of observation, indicating the utility of vascular smooth muscle cells for gene therapy in humans.

Full text

PDF
1138

Images in this article

1140Image
on p.1140
1140Image
on p.1140
1140Image
on p.1140
1140Image
on p.1140
1140Image
on p.1140
1140Image
on p.1140
1140Image
on p.1140
1141Image
on p.1141

Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Adam M. A., Ramesh N., Miller A. D., Osborne W. R. Internal initiation of translation in retroviral vectors carrying picornavirus 5' nontranslated regions. J Virol. 1991 Sep;65(9):4985–4990. doi: 10.1128/jvi.65.9.4985-4990.1991. [DOI] [PMC free article] [PubMed] [Google Scholar]
  2. Clowes A. W., Collazzo R. E., Karnovsky M. J. A morphologic and permeability study of luminal smooth muscle cells after arterial injury in the rat. Lab Invest. 1978 Aug;39(2):141–150. [PubMed] [Google Scholar]
  3. Clowes A. W., Reidy M. A., Clowes M. M. Kinetics of cellular proliferation after arterial injury. I. Smooth muscle growth in the absence of endothelium. Lab Invest. 1983 Sep;49(3):327–333. [PubMed] [Google Scholar]
  4. Dichek D. A., Nussbaum O., Degen S. J., Anderson W. F. Enhancement of the fibrinolytic activity of sheep endothelial cells by retroviral vector-mediated gene transfer. Blood. 1991 Feb 1;77(3):533–541. [PubMed] [Google Scholar]
  5. Felton J., Meisler M., Paigen K. A locus determining beta-galactosidase activity in the mouse. J Biol Chem. 1974 May 25;249(10):3267–3272. [PubMed] [Google Scholar]
  6. Hershfield M. S., Buckley R. H., Greenberg M. L., Melton A. L., Schiff R., Hatem C., Kurtzberg J., Markert M. L., Kobayashi R. H., Kobayashi A. L. Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase. N Engl J Med. 1987 Mar 5;316(10):589–596. doi: 10.1056/NEJM198703053161005. [DOI] [PubMed] [Google Scholar]
  7. Hock R. A., Miller A. D., Osborne W. R. Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell lines. Blood. 1989 Aug 1;74(2):876–881. [PubMed] [Google Scholar]
  8. Lim C. S., Chapman G. D., Gammon R. S., Muhlestein J. B., Bauman R. P., Stack R. S., Swain J. L. Direct in vivo gene transfer into the coronary and peripheral vasculatures of the intact dog. Circulation. 1991 Jun;83(6):2007–2011. doi: 10.1161/01.cir.83.6.2007. [DOI] [PubMed] [Google Scholar]
  9. Lombardi D. M., Reidy M. A., Schwartz S. M. Methodologic considerations important in the accurate quantitation of aortic smooth muscle cell replication in the normal rat. Am J Pathol. 1991 Feb;138(2):441–446. [PMC free article] [PubMed] [Google Scholar]
  10. Miller A. D., Buttimore C. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol Cell Biol. 1986 Aug;6(8):2895–2902. doi: 10.1128/mcb.6.8.2895. [DOI] [PMC free article] [PubMed] [Google Scholar]
  11. Miller A. D. Progress toward human gene therapy. Blood. 1990 Jul 15;76(2):271–278. [PubMed] [Google Scholar]
  12. Miller A. D., Rosman G. J. Improved retroviral vectors for gene transfer and expression. Biotechniques. 1989 Oct;7(9):980-2, 984-6, 989-90. [PMC free article] [PubMed] [Google Scholar]
  13. Miller A. D., Trauber D. R., Buttimore C. Factors involved in production of helper virus-free retrovirus vectors. Somat Cell Mol Genet. 1986 Mar;12(2):175–183. doi: 10.1007/BF01560664. [DOI] [PubMed] [Google Scholar]
  14. Miller D. G., Adam M. A., Miller A. D. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol. 1990 Aug;10(8):4239–4242. doi: 10.1128/mcb.10.8.4239. [DOI] [PMC free article] [PubMed] [Google Scholar]
  15. Nabel E. G., Plautz G., Boyce F. M., Stanley J. C., Nabel G. J. Recombinant gene expression in vivo within endothelial cells of the arterial wall. Science. 1989 Jun 16;244(4910):1342–1344. doi: 10.1126/science.2499928. [DOI] [PubMed] [Google Scholar]
  16. Nabel E. G., Plautz G., Nabel G. J. Site-specific gene expression in vivo by direct gene transfer into the arterial wall. Science. 1990 Sep 14;249(4974):1285–1288. doi: 10.1126/science.2119055. [DOI] [PubMed] [Google Scholar]
  17. Osborne W. R. Human red cell purine nucleoside phosphorylase. Purification by biospecific affinity chromatography and physical properties. J Biol Chem. 1980 Aug 10;255(15):7089–7092. [PubMed] [Google Scholar]
  18. Osborne W. R., Miller A. D. Design of vectors for efficient expression of human purine nucleoside phosphorylase in skin fibroblasts from enzyme-deficient humans. Proc Natl Acad Sci U S A. 1988 Sep;85(18):6851–6855. doi: 10.1073/pnas.85.18.6851. [DOI] [PMC free article] [PubMed] [Google Scholar]
  19. Osborne W. R., Spencer N. Partial purification and properties of the common inherited forms of adenosine deaminase from human erythrocytes. Biochem J. 1973 May;133(1):117–123. doi: 10.1042/bj1330117. [DOI] [PMC free article] [PubMed] [Google Scholar]
  20. Palmer T. D., Hock R. A., Osborne W. R., Miller A. D. Efficient retrovirus-mediated transfer and expression of a human adenosine deaminase gene in diploid skin fibroblasts from an adenosine deaminase-deficient human. Proc Natl Acad Sci U S A. 1987 Feb;84(4):1055–1059. doi: 10.1073/pnas.84.4.1055. [DOI] [PMC free article] [PubMed] [Google Scholar]
  21. Palmer T. D., Rosman G. J., Osborne W. R., Miller A. D. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci U S A. 1991 Feb 15;88(4):1330–1334. doi: 10.1073/pnas.88.4.1330. [DOI] [PMC free article] [PubMed] [Google Scholar]
  22. Palmer T. D., Thompson A. R., Miller A. D. Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B. Blood. 1989 Feb;73(2):438–445. [PubMed] [Google Scholar]
  23. Pelletier J., Sonenberg N. Internal initiation of translation of eukaryotic mRNA directed by a sequence derived from poliovirus RNA. Nature. 1988 Jul 28;334(6180):320–325. doi: 10.1038/334320a0. [DOI] [PubMed] [Google Scholar]
  24. Plautz G., Nabel E. G., Nabel G. J. Introduction of vascular smooth muscle cells expressing recombinant genes in vivo. Circulation. 1991 Feb;83(2):578–583. doi: 10.1161/01.cir.83.2.578. [DOI] [PubMed] [Google Scholar]
  25. Rosenberg S. A., Aebersold P., Cornetta K., Kasid A., Morgan R. A., Moen R., Karson E. M., Lotze M. T., Yang J. C., Topalian S. L. Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med. 1990 Aug 30;323(9):570–578. doi: 10.1056/NEJM199008303230904. [DOI] [PubMed] [Google Scholar]
  26. Sanes J. R., Rubenstein J. L., Nicolas J. F. Use of a recombinant retrovirus to study post-implantation cell lineage in mouse embryos. EMBO J. 1986 Dec 1;5(12):3133–3142. doi: 10.1002/j.1460-2075.1986.tb04620.x. [DOI] [PMC free article] [PubMed] [Google Scholar]
  27. Tsukada T., Tippens D., Gordon D., Ross R., Gown A. M. HHF35, a muscle-actin-specific monoclonal antibody. I. Immunocytochemical and biochemical characterization. Am J Pathol. 1987 Jan;126(1):51–60. [PMC free article] [PubMed] [Google Scholar]
  28. Wei C. M., Gibson M., Spear P. G., Scolnick E. M. Construction and isolation of a transmissible retrovirus containing the src gene of Harvey murine sarcoma virus and the thymidine kinase gene of herpes simplex virus type 1. J Virol. 1981 Sep;39(3):935–944. doi: 10.1128/jvi.39.3.935-944.1981. [DOI] [PMC free article] [PubMed] [Google Scholar]
  29. Wilson J. M., Birinyi L. K., Salomon R. N., Libby P., Callow A. D., Mulligan R. C. Implantation of vascular grafts lined with genetically modified endothelial cells. Science. 1989 Jun 16;244(4910):1344–1346. doi: 10.1126/science.2734614. [DOI] [PubMed] [Google Scholar]

Articles from Proceedings of the National Academy of Sciences of the United States of America are provided here courtesy of National Academy of Sciences

RESOURCES