Fetal gene transfer by transuterine injection of cationic liposome–DNA complexes (original) (raw)
References
Ledley, F.D. Prenatal application of somatic gene therapy. Obstet Gynecol. Clin. North Am.3, 611–620 ( 1993). Google Scholar
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). CASPubMed Google Scholar
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. Blood78, 1132– 1139 (1991). CASPubMed Google Scholar
Pitt, B.R. et al. Retrovirus-mediated gene transfer in lungs of living fetal sheep. Gene Ther.5, 344–350 ( 1995). Google Scholar
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
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). ArticleCAS Google Scholar
Fleischman, R.A., Custer, R.P. & Mintz B. Totipotent hematopoietic stem cells: normal self-renewal and differentiation after transplantation between mouse fetuses. Cell30, 351– 359 (1982). ArticleCAS Google Scholar
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. USA86, 7456–7459 ( 1989). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
Hyde, S.C. et al. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature362, 250– 255 (1993). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
Nabel, E.G., Plautz, G. & Nabel, G.J. Site-specific gene expression in vivo by direct gene transfer into the arterial wall. Science249, 1285 –1288 (1990). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
Zhu, N., Liggitt, D., Liu, Y. & Debs, R. Systemic gene expression after intravenous DNA delivery into adult mice. Science261, 209–211 (1993). ArticleCAS Google Scholar
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). CASPubMed Google Scholar
Kawakami, M. et al. Levels of serum granulocyte colony-stimulating factor in patients with infections. Blood76, 1962– 1964 (1990). CASPubMed Google Scholar
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). CASPubMed Google Scholar
Liu, Y. et al. Cationic liposome-mediated intravenous gene delivery in mice. J. Biol. Chem.27, 24864–24870 (1995). Article Google Scholar
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). ArticleCAS Google Scholar
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. USA93, 3056–3061 (1996). ArticleCAS Google Scholar
Thierry, A.R. et al. Systemic gene therapy: biodistribution and long-term expression of a transgene in mice. Proc. Natl Acad. Sci. USA92, 9742–9746 (1995). ArticleCAS Google Scholar
Cooper, M.J. et al. Safety-modified episomal vectors for human gene therapy. Proc. Natl. Acad. Sci. USA94, 6450– 6455 (1997). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
Templeton, N.S. et al. Improved DNA: liposome complexes for increased systemic delivery and gene expression. Nat. Biotechnol.15, 647–652 (1997). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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
Yang, Y. et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA91, 4407–4411 (1994). ArticleCAS Google Scholar
Sekhon, H.S. & Larson, J.E. In utero gene transfer into the pulmonary epithelium. Nat. Med.1, 1201– 1203 (1995). ArticleCAS Google Scholar
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. Lancet349, 619– 620 (1997). ArticleCAS Google Scholar
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). CASPubMedPubMed Central Google Scholar
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. USA95, 11377– 11382 (1998). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
Low, J.A. The relationship of asphyxia in the mature fetus to long-term neurologic function. Clin. Obstet. Gynecol.36, 82– 90 (1993). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
Kurth, R. Risk potential of the chromosomal insertion of foreign DNA. Ann. NY Acad. Sci.772, 140–151 (1995). ArticleCAS Google Scholar
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
Solodin, I. et al. A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery. Biochemistry34, 13537–13544 (1995). ArticleCAS Google Scholar
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
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. Acta557, 9–20 ( 1979). ArticleCAS Google Scholar
Johnston, R.F., Pickett, S.L. & Barker, D.L. Autoradiography using storage phosphor technology. Electrophoresis11, 355– 360 (1990). ArticleCAS Google Scholar
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). ArticleCAS Google Scholar
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. USA90, 11381–11385 (1993). ArticleCAS Google Scholar