Fetal gene transfer by transuterine injection of cationic liposome–DNA complexes (original) (raw)

References

  1. Ledley, F.D. Prenatal application of somatic gene therapy. Obstet Gynecol. Clin. North Am. 3, 611–620 ( 1993).
    Google Scholar
  2. Hatzoglou, M. et al. Hepatic gene transfer in animals using retroviruses containing the promoter from the gene for phosphoenolpyruvate carboxykinase. J. Biol. Chem. 265, 17285–17293 (1990).
    CAS PubMed Google Scholar
  3. Clapp, D.W., Dumenco, L.L., Hatzoglou, M. & Gerson, S.L. Fetal liver hematopoietic stem cells as a target for in utero retroviral gene transfer. Blood 78, 1132– 1139 (1991).
    CAS PubMed Google Scholar
  4. Pitt, B.R. et al. Retrovirus-mediated gene transfer in lungs of living fetal sheep. Gene Ther. 5, 344–350 ( 1995).
    Google Scholar
  5. Hatzoglou, M., Moorman, A. & Lamers, W. Persistent expression of genes transferred in the fetal rat liver via retroviruses. Somat. Cell Mol. Genet. 4, 265–278 (1995).
    Article Google Scholar
  6. Kay, M.A. et al. Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum. Gene Ther. 3, 641–647 (1992).
    Article CAS Google Scholar
  7. Fleischman, R.A., Custer, R.P. & Mintz B. Totipotent hematopoietic stem cells: normal self-renewal and differentiation after transplantation between mouse fetuses. Cell 30, 351– 359 (1982).
    Article CAS Google Scholar
  8. Toles, J.F., Chui, D.H., Belbeck, L.W., Starr, E. & Barker, J.E. Hematopoietic stem cells in murine embryonic yolk sac and peripheral blood. Proc. Natl. Acad. Sci. USA 86, 7456–7459 ( 1989).
    Article CAS Google Scholar
  9. Debs, R. et al. Extended, high level transgene expression in rodent lung cells ex vivo. Am. J. Respir. Cell. Mol. Biol. 7, 406– 413 (1992).
    Article CAS Google Scholar
  10. Tsukamoto, M., Ochiya, T., Yoshida, S., Sugimura, T. & Terada M. Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice. Nat. Genet. 9, 243–248 (1995).
    Article CAS Google Scholar
  11. Alton, E,W. et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat. Genet. 5, 135–142 (1993).
    Article CAS Google Scholar
  12. Hyde, S.C. et al. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 362, 250– 255 (1993).
    Article CAS Google Scholar
  13. Canonico, A.E., Conary, J.T., Meyrick, B.O. & Brigham, K.L. Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits. Am. J. Respir. Cell. Mol. Biol. 10 , 24–29 (1994).
    Article CAS Google Scholar
  14. Sorscher, E.J. et al. Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: a phase I trial of safety and efficacy in the nasal airway. Hum. Gene Ther. 5, 1259– 1277 (1994).
    Article CAS Google Scholar
  15. Nabel, E.G., Plautz, G. & Nabel, G.J. Site-specific gene expression in vivo by direct gene transfer into the arterial wall. Science 249, 1285 –1288 (1990).
    Article CAS Google Scholar
  16. McCray, P.B. et al. Adenoviral-mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo. J. Clin. Invest. 95 , 2620–2631 (1995).
    Article CAS Google Scholar
  17. Zhu, N., Liggitt, D., Liu, Y. & Debs, R. Systemic gene expression after intravenous DNA delivery into adult mice. Science 261, 209–211 (1993).
    Article CAS Google Scholar
  18. Philip, R., Liggitt, D., Philip, M., Dazin, P. & Debs, R.J. In vivo gene delivery: efficient transfection of T lymphocytes in adult mice. J. Biol Chem. 268, 16087– 16090 (1993).
    CAS PubMed Google Scholar
  19. Kawakami, M. et al. Levels of serum granulocyte colony-stimulating factor in patients with infections. Blood 76, 1962– 1964 (1990).
    CAS PubMed Google Scholar
  20. Ichikawa, T., Kuwaki, T., Tachibana, K., Yanagida, M. & Matsuki, S. A highly sensitive enzyme immunoassay for G-CSF in human plasma. Exp. Hematol. 23, 192–195 (1995).
    CAS PubMed Google Scholar
  21. Liu, Y. et al. Cationic liposome-mediated intravenous gene delivery in mice. J. Biol. Chem. 27, 24864–24870 (1995).
    Article Google Scholar
  22. Gilgenkrantz, H. et al. Transient expression of genes transferred in vivo into heart using first-generation adenoviral vectors: role of the immune response. Hum. Gene Ther. 6, 1265–1274 (1995).
    Article CAS Google Scholar
  23. Walter, J., You, Q., Hagstrom, JN., Sands, M. & High, K.A. Successful expression of human factor IX following repeat administration of adenoviral vector in mice. Proc. Natl. Acad. Sci. USA 93, 3056–3061 (1996).
    Article CAS Google Scholar
  24. Thierry, A.R. et al. Systemic gene therapy: biodistribution and long-term expression of a transgene in mice. Proc. Natl Acad. Sci. USA 92, 9742–9746 (1995).
    Article CAS Google Scholar
  25. Cooper, M.J. et al. Safety-modified episomal vectors for human gene therapy. Proc. Natl. Acad. Sci. USA 94, 6450– 6455 (1997).
    Article CAS Google Scholar
  26. Hong, K., Zheng, W., Baker, A., & Papahadjopoulos, D. Stabilization of cationic liposome-plasmid DNA complexes by polyamines and poly(ethylene glycol)-phospholipid conjugates for efficient in vivo gene delivery. FEBS Lett. 400, 233–237 (1997).
    Article CAS Google Scholar
  27. Templeton, N.S. et al. Improved DNA: liposome complexes for increased systemic delivery and gene expression. Nat. Biotechnol. 15, 647–652 (1997).
    Article CAS Google Scholar
  28. Stewart, M.J. et al. Gene transfer in vivo with DNA–liposome complexes: safety and acute toxicity in mice. Hum. Gene Ther. 3, 267–275 (1992).
    Article CAS Google Scholar
  29. Canonico, A.E., Plitman, J.D., Conary, J.T., Meyrick, B.O. & Brigham K.L. No lung toxicity after repeated aerosol or intravenous delivery of plasmid–cationic liposome complexes. J. Appl. Physiol. 77, 415– 419 (1994).
    Article CAS Google Scholar
  30. Vincent, M.C. et al. Adenovirus-mediated gene transfer to the respiratory tract of fetal sheep in utero. Hum. Gene Ther. 8, 1019– 1028 (1995).
    Article Google Scholar
  31. Yang, Y. et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA 91, 4407–4411 (1994).
    Article CAS Google Scholar
  32. Sekhon, H.S. & Larson, J.E. In utero gene transfer into the pulmonary epithelium. Nat. Med. 1, 1201– 1203 (1995).
    Article CAS Google Scholar
  33. Larson, J.E., Morrow, S.L., Happel, L., Sharp, J.F. & Cohen, J.C. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 349, 619– 620 (1997).
    Article CAS Google Scholar
  34. Halbert, C.L. et al. Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. J. Virol. 71, 5932–5941 (1997).
    CAS PubMed PubMed Central Google Scholar
  35. Kafri, T. et al. Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: implications for gene therapy. Proc. Natl. Acad. Sci. USA 95, 11377– 11382 (1998).
    Article CAS Google Scholar
  36. Iwamoto, H.S., Trapnell B.C., McConnell C.J., Daugherty, C. & Whitsett, J.A. Pulmonary inflammation associated with repeated, prenatal exposure to an E1, E3-deleted adenoviral vector in sheep. Gene Ther. 6, 98– 106 (1999).
    Article CAS Google Scholar
  37. Ketteler, M., Noble, N.A. & Border, W.A. Increased expression of transforming growth factor-beta in renal disease. Curr. Opin. Nephrol. Hypertens. 3 , 446–452 (1994).
    Article CAS Google Scholar
  38. Bartke, A. et al. Neuroendocrine and reproductive consequences of overexpression of growth hormone in transgenic mice. Proc. Soc. Exp. Biol. Med. 206, 345–359 (1994).
    Article CAS Google Scholar
  39. Holtzman, D.M., Sheldon, R.A., Jaffe, W., Cheng, Y. & Ferriero, D.M. Nerve growth factor protects the neonatal brain against hypoxic-ischemic injury. Ann. Neurol. 39, 114–122 (1996).
    Article CAS Google Scholar
  40. Low, J.A. The relationship of asphyxia in the mature fetus to long-term neurologic function. Clin. Obstet. Gynecol. 36, 82– 90 (1993).
    Article CAS Google Scholar
  41. Paneth, N. & Stark, R.I. Cerebral palsy and mental retardation in relation to indicators of perinatal asphyxia. An epidemiologic overview. Am. J. Obstet. Gynecol. 147, 960– 966 (1983).
    Article CAS Google Scholar
  42. Mason, C.A. et al. Gene transfer in utero biologically engineers a patent ductus arteriosus in lambs by arresting fibronectin-dependent neointimal formation. Nat. Med. 5, 176–182 ( 1999).
    Article CAS Google Scholar
  43. Kurth, R. Risk potential of the chromosomal insertion of foreign DNA. Ann. NY Acad. Sci. 772, 140–151 (1995).
    Article CAS Google Scholar
  44. Sambrook, J., Fritsch, E.F. & Maniatis, T. in Molecular cloning: a laboratory manual 2nd edn (eds Ford, N., Nolan, C. & Ferguson, M.) (Cold Spring Harbor Laboratory Press, Cold Spring Harbor, New York; 1989).
    Google Scholar
  45. Solodin, I. et al. A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery. Biochemistry 34, 13537–13544 (1995).
    Article CAS Google Scholar
  46. Mounkes, L. et al. In vivo transfection of animals by intravenous injection of cationic liposome:DNA complexes. J. Lipid Res. 7, 161–176 (1997).
    CAS Google Scholar
  47. Olson, F., Hunt, C.A., Szoka, F.C., Vail, W.J. & Papahadjopoulos, D. Preparation of liposomes of defined size distribution by extrusion through polycarbonate membranes. Biochim. Biophys. Acta 557, 9–20 ( 1979).
    Article CAS Google Scholar
  48. Johnston, R.F., Pickett, S.L. & Barker, D.L. Autoradiography using storage phosphor technology. Electrophoresis 11, 355– 360 (1990).
    Article CAS Google Scholar
  49. Bout, A., Valerio, D. & Scholte, B.J. In vivo transfer and expression of the lacZ gene in the mouse lung. Exp. Lung Res. 19, 193– 202 (1993).
    Article CAS Google Scholar
  50. Gaensler, K.M., Kitamura, M. & Kan, Y.W. Germ-line transmission and developmental regulation of a 150-kb yeast artificial chromosome containing the human beta-globin locus in transgenic mice. Proc. Natl Acad. Sci. USA 90, 11381–11385 (1993).
    Article CAS Google Scholar

Download references