Gene therapy for genetic diseases

Pediatrics International

Volumn 40, Issue 3, 1998, Pages 191-203

  Desnick, Robert J ^a   Schuchman, Edward H ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

DNA protein conjugates; Gene therapy; Gene transfer; Liposomes; Viral vectors

Indexed keywords

COMPLEMENTARY RNA; PLASMID VECTOR; RIBOZYME; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; ARTICLE; EXPRESSION VECTOR; GENE INSERTION; GENE TARGETING; GENE THERAPY; HEMATOLOGIC DISEASE; HERPES SIMPLEX VIRUS; HUMAN; LIVER DISEASE; NEUROLOGIC DISEASE; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS;

CENTRAL NERVOUS SYSTEM DISEASES; FEMALE; FETAL DISEASES; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LIPOSOMES; LIVER DISEASES; PREGNANCY;

EID: 0031866965     PISSN: 13288067     EISSN: 1442200X     Source Type: Journal    
DOI: 10.1111/j.1442-200X.1998.tb01912.x     Document Type: Article

References (80)

1. Weiss R., Teich N., Varmus H., Coffin J. (eds.). RNA Tumor Viruses. Cold Spring Harbor, New York, 1984.
2. Hu Ws, Temin Hm. Retroviral recombination and reverse transcription. Science 1990; 250: 1227–33.
3. Gilboa E. Retroviral gene transfer: Applications to human therapy. Prog. Clin. Biol. Res. 1990; 352: 301–11.
4. Miller Da. Human gene therapy comes of age. Nature 1992; 357: 455–60.
5. Mulligan Rc. The basic science of gene therapy. Science 1993; 260: 926–32.
6. Kozarsky Kf, Wilson Jm. Gene therapy: adenovirus vectors. Curr. Opin. Genet. Dev. 1993; 3: 499–503.
7. Rosenfeld M., Yoshimura K., Tranpnell B. et al. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 1992; 68: 143–55.
8. Yang Y., Nunes Fa, Berencsi K., Furth Ee, Gonczol E., Wilson Jm. Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc. Natl Acad. Sci. USA 1994; 91: 4407–11.
9. Crystal Rg, Mcelvaney Ng, Rosenfeld Ma et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nature Genet. 1994;8: 42–51.
10. Knowles Mr, Hohneker Kw, Zhou Z. et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N. Engl. J. Med. 1995; 333: 823–30.
11. Mcloughlin Sk, Labow Ma, Wright R., Bems Ki. Adeno-associated virus general transduction vectors: Analysis of proviral structures. J. Virol. 1984; 62: 1963–9.
12. Siegl G., Bates Rc, Berns Ki et al. Characteristics and taxonomy of Parvoviridae. Intervirology 1985; 23: 61–73.
13. Kohn Rm, Siniscalco M., Samulski Rj et al. Site-specific integration by adeno-associated virus. Proc. Natl Acad. Sci. USA 1990;87: 2211–15.
14. Breakefield Xo. Gene delivery into the brain using virus vectors. Nature Genet. 1993; 3: 187–9.
15. Wolfe Jh. Sands Ms, Barker Je et al. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 1992: 360: 749–53.
16. Feigner Pl. Rhodes G. Gene therapeutics. Nature 1991; 349: 351–2.
17. Smith Jg. Walzem Rl. German Jb. Liposomes as agents of DNA transfer. Biochim. Biophys. Acta 1993; 1154: 327–40.
18. Alving Cr. Steck Ea. Chapman Wl et al. Therapy of leishmaniasis: Superior efficacies of liposome-encapsulated drugs. Proc: Natl Acad. Sci. USA 1978; 75: 2959–63.
19. Ostro Mj. Liposomes. Sci. Am. 1987: 256: 102–11.
20. Behr Jr DNA strongly binds to micelles and vesicles containing lipopolyamines or lipointercalants. Tetrahedron Lett. 1986; 27: 5861–4.
21. Feigner Pl Gadek Tr. Holm M. et al. Lipofection: A highly efficient lipid mediated DNA transfection procedure. Proc. Natl Acad. Sci. USA 1987; 84: 7413–17.
22. Wang Cy. Huang L. Plasmid DNA adsorbed to pH-sensitive liposomes efficiently transforms the target cells. Biochem. Biophys. Res. Comm. 1987; 147: 980–5.
23. Wang Cy. Huang L. Highly efficient DNA delivery mediated by pH-sensitive immunoliposomes. Biochemistry 1989: 28: 9508–14.
24. Lasic Dd. Martin Fj. Gabizon A. Huang Sk, Papahadjopoulos D. Sterically stabilized liposomes: A hypothesis on the molecular origin of the extended circulation times. Biochim. Biophys. Acta 1991; 1070: 187–92.
25. Wolff Ja Malone Rw. Williams P. et al. Direct gene transfer into mouse muscle in vivo. Science 1990; 247: 1465–8.
26. Danko I., Fritz Jd, Latendresse Js, Herweijer H., Schultz E. Wolff Ja. Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection. Hum Mol. Gene 1993; 2: 2055–61.
27. Michael Si, Curiel Dt. Strategies to achieve targeted gene delivery via receptor-mediated endocytosis pathway. Gene Then 1994:1: 223–32.
28. Chowdhury Mr. Wu Ch, Wu Gy. Yemeni Pc, Bommineni Vr, Chowdhury Jr. Fate of DNA targeted of the liver by asialoglycoprotein receptor-mediated endocytosis in vivo J. Biol Chem. 1993: 268: 11265–71.
29. Wu Gy, Wu Ch. Receptor-mediated gene delivery and expression in vivo. J. Biol. Chem. 1988; 263: 14 621–4.
30. Wagner E., Zenke M. Cotten M. Beug J., Birnstiel Ml. Trans-ferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl Acad. Sci. USA 1990; 87: 3410–14.
31. Ferkol. T., Kaetzel Cs, Davis Pb. Gene transfer into respiratory epithelial cells by targeting the polymeric immunoglobulin receptor. J. Clin. Invest. 1993; 92: 2394–400.
32. Curiel Dt, Wagner E., Cotten M. et al. High efficiency gene transfer mediated by adenovirus coupled to DNA polylysine complexes. Hum. Gene Then 1992; 3: 147–54.
33. Cristiano Rj, Smith L., Woo Slc. Hepatic gene therapy: Adenovirus enhancement of receptor-mediated gene delivery and expression in primary hepatocytes. Proc. Natl Acad. Sci. USA 1993; 90: 2122–6.
34. Cotten M., Wagner E. Zatloukal K., Phillips S., Curiel Dt, Brin-Stiel Ml. High efficiency receptor mediated delivery of small and large (48 kilobase) gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles. Proc. Natl Acad. Sci. USA 1992; 89: 6094–8.
35. Boulikas T. Nuclear localization signals (NLS). Crit. Rev. Eurkaryot. Gene Expr. 1993: 3: 193–227.
36. Southern Pj, Berg P. Transformation of mammalian cells to antibiotic resistance with a bacterial gene under control of the SV40 early region promoter. J. Mol. Appl. Genet. 1982; 1: 327–41.
37. Foecking Mk, Hofstetter H. Powerful and versatile enhancer-promoter unit for mammalian expression vectors. Gene 1986; 45: 101–5.
38. Culver Kw, Ram Z., Wallbridge S., Ishii H., Oldfield Eh, Blaese Rm. In vivo gene transfer with retroviral vector-producer cells for treatment of experimental brain tumors. Science 1992; 256: 1550–2.
39. Ho M. Characteristics of cytomegalovirus. In: Ho M. (ed.). Cytomegalovirus: Biology and Infection. Plenum Press, New York, 1991: 7–34.
40. Mclachlin Jr, Mittereder N., Daucher Mb. Kadan M. Eglitis Ma. Factors affecting retroviral vector function and structural integrity. Virology 1993: 195: 1–5.
41. Gow A., Friedrich Vl, Lazzarini Ra. Myelin basic protein gene contains separate enhancers for oligodendrocyte and Schwann cell expression. J. Cell Biol. 1992: 119: 605–16.
42. Karlsson S. Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood 1991; 78: 2481–92.
43. Bodine Dm, Karlsson S., Nienhuis Aw. Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. Proc. Natl Acad. Sci. USA 1989 8897–901.
44. Schuening Fg, Kawahara K., Miller Ad et al. Retrovirus mediated gene transduction into long term repopulating marrow cells of dogs. Blood 1991; 78: 2568–73.
45. Broxmeyer He, Douglas Gw, Hangoc G. et al. Human umbilical cord blood as a source of transplantable hematopoietic stem and progenitor cells. Curt: Top. Microbiol. Immunol. 1992: 177: 195–204.
46. Eaves Cj, Sutherland Hj, Udomsakdi C. et al. The human hematopoietic stem cell in vitro and in vivo. Blood Cells 1992; 18: 301–7.
47. Berenson Rj. Transplantation of CD34 + hematopoietic precursors: clinical rationale. Transplant. Proc. 1992; 24: 3032–4.
48. Migliaccio G., Migliaccio Ar, Druzin Ml, Giardina P-Jv, Zsebo Km, Adamson Jw. Long term generation of colony forming cells in liquid culture of CD34 + cord blood cells in the presence of recombinant human stem cell factor. Blood 1992;79: 2620–7.
49. Bernardi Ac, Wang A., Levine Jd, Lopez P., Scadden Dt. Functional isolation and characterization of human hematopoietic stem cells. Science 1995; 267: 104–8.
50. Kay Ma, Woo Slc. Gene therapy for metabolic diseases. Trends Genet. 1994; 10: 253–7.
51. Ponder K., Gupta S., Leland F. et al. Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation. Proc. Natl Acad. Sci. USA 1991; 88: 1217–21.
52. Miyazaki et al.
53. Fujiwara et al. Stimulation of liver growth by exogenous human hepatocyte growth factor in normal and partially hepa-tectomized rats. Hepatology 1994; 18: 1443–9.
54. Li Q., Kay Ma, Finegold M., Stratford-Perricaudet Ld, Woo Sl. Assessment of recombinant adenoviral vectors for hepatic gene therapy. Hum. Gene Ther. 1993; 4: 403–9.
55. Herz J., Gerard Rd. Adenovirus-mediated transfer of the low density lipoprotein receptor gene acutely accelerates cholesterol clearance in normal mice. Proc. Natl Acad. Sci. USA 1993;90: 2812–16.
56. Kozarsky Kf, Mckinley Dr, Austin Ll, Raper Se, Stratford-Perricaudet Ld, Wilson Jm. In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenovirus. J. Biol, Chem. 1994:269: 13695–702.
57. Suhr St, Gage Fh. Gene therapy for neurologic diseases. Arch. Neurol. 1993; 50: 1252–68.
58. Friedmann T. Gene therapy for neurologic disorders. Trends Genet. 1994; 10: 310–14.
59. Schuchman Eh, Loannou Ya, Rattazzi Mc, Desnick Rj. Neural gene therapy for inherited diseases with mental retardation: Principles and prospects. MRDD Res. Rev. 1995; 1: 39–48.
60. Fabry Z., Raine Cs, Hart Mn. Nervous tissue as an immune compartment: the dialect of the immune response in the CNS. Immunol, Today 1994; 15: 218–24.
61. Snyder Ey, Taylor Rm, Wolfe Jh. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 1995; 374: 367–70.
62. Hickey Wf, Kimura H. Perivascular microglial cells of the CNS are bone marrow derived and present antigen. Science 1988;239: 290–2.
63. Culver Kw, Anderson Wf, Blaese Rm. Lymphocyte gene therapy. Hum. Gene Ther. 1991; 2: 107–9.
64. Blaese Rm, Culver Kw, Miller D. et al. T-lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after four years. Science 1995; 270: 475–80.
65. Bordignon C., Notarangelo Ld, Nobili N. et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 1995; 270: 470–5.
66. Kohn Db, Weinberg Ki, Lenarsky C. et al. Gene therapy for neonates with ADA deficiency by transfer of the human ADA cDNA into umbilical cord CD34 + cells: Two year follow up. Pediatr. Res. 1995; 37: A9.
67. Colledge Wh. Cystic fibrosis gene therapy. Curr. Opin. Genet. Dew 1994; 4: 466–71.
68. Grossman M., Raper Se, Kozarsky K. et al. Successful ex vivo gene therapy directed to liver in a patient with familial hyper-cholesterolaemia. Nature Genet. 1994; 6: 335–41.
69. Srour E., Zanjani Ed, Brandt Je et al. Sustained human hematopoiesis in sheep transplanted in utero during early gestation with fractionated adult human bone marrow cells. Blood 1992; 79: 1404–12.
70. Roodman Gd, Kuehl Tj, Vandeberg Jl, Muirhead Dy. In utero bone marrow transplantation of fetal baboons with mismatched adult baboon marrow. Blood Cells 1991; 17: 367–75.
71. Pallavicini Mg, Flake Aw, Madden D. et al. Hemopoietic chimerism in rodents transplanted in utero with fetal human hemopoietic cells. Transplant. Proc. 1992; 24: 524–43.
72. Touraine Jl, Raudrant D., Royo C. et al. In utero transplantation of hemopoietic stem cells in humans. Transplant. Proc. 1991:23: 1706–8.
73. Tsukamoto M., Ochiya T., Yoshida S., Sugimura T., Terada M. Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice. Nature Genet. 1995; 9: 243–8.
74. Jahner D., Haase K., Mulligan, R., Jaenisch R. Insertion of the bacterial gpt gene into the germ line of mice by retroviral infection. Proc. Natl Acad. Sci. USA 1985; 82: 6927–31.
75. Altaian S. RNA-enzyme directed gene therapy. Proc. Natl Acad. Sci. USA 1993; 90: 10898–900.
76. Yu M., Ojwang J., Yamada O. et al. A hairpin ribozyme inhibits expression of diverse strains of human immunodeficiency virus type 1. Proc. Natl Acad. Sci. USA 1993; 90: 6340–4.
77. Cantor Gh, Mcelwain Tf, Birkebak Ta, Palmer Gh. Ribozyme cleaves rex/tax mRNA and inhibits bovine leukemia virus expression. Proc. Natl Acad. Sci. USA 1993; 90: 10932–6.
78. Ohkawa J., Yuyama N., Takebe Y., Nishikawa S., Taira K. Importance of independence in ribozyme reactions: Kinetic behavior of trimmed and of simply connected ribozymes with potential activity against human immunodeficiency virus. Proc. Natl Acad. Sci. USA 1993; 90: 11302–6.
79. Thompson Jd, Macejak D. Couture L., Stinchomb Dt. Ribozymes in gene therapy. Nat. Med. 1995; 1: 277–8.
80. Bollag Aj, Waldman As, Liskay Rm. Homologous recombination in mammalian cells. Ann. Rev. Genet. 1989; 23: 199–225.