Receptor ligand-facilitated cationic liposome delivery of anti-HIV-1 Rev-binding aptamer and ribozyme DNAs

Journal of Drug Targeting

Volumn 5, Issue 4, 1998, Pages 247-259

  Konopka, Krystyna ^a   Düzgüneş, Nejat ^a,b   Rossi, John ^c   Lee, Nan Sook ^c  

^a UNIVERSITY OF THE PACIFIC   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

Author keywords

Aptamer; Human immunodeficiency virus (HIV); Lipofectin; Ribozyme; Transfection; Transferrin

Indexed keywords

ANTI HUMAN IMMUNODEFICIENCY VIRUS AGENT; ANTISENSE OLIGONUCLEOTIDE; ANTIVIRUS AGENT; BINDING PROTEIN; GAG PROTEIN; LIPOFECTIN; LIPOSOME; REV PROTEIN; RIBOZYME; UNCLASSIFIED DRUG; VIRUS DNA; VIRUS PROTEIN;

ARTICLE; CELL VIABILITY; CONTROLLED STUDY; DNA TRANSFECTION; DRUG DELIVERY SYSTEM; GENE EXPRESSION; GENE TARGETING; HELA CELL; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS; PRIORITY JOURNAL; PROMOTER REGION;

EID: 0031691988     PISSN: 1061186X     EISSN: None     Source Type: Journal    
DOI: 10.3109/10611869808995879     Document Type: Article

References (47)

1. + hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits human immunodeficiency virus type 1 replication in myelomonocytic cells produced in long-term culture. J. Virol., 70, 4352-4360.
2. Bertrand E., Castanotto D., Zhou C., Carbonnelle C., Lee N.S. , Good P., Chatterjee S., Grange T., Pictet R., Kohn D., Engelke D. and Rossi J.J. (1997) The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization. RNA 3, 75-88.
3. Bridges S.H. and Sarver N. (1995) Gene therapy and immune restoration for HIV disease. Lancet, 345, 427-431.
4. Brigham K.L., Meyrick B., Christman B., Magnuson M., King G. and Berry L.C.Jr. (1989) In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci., 298, 278-281.
5. Cheng P.-W. (1996) Receptor ligand-facilitated gene transfer: enhancement of liposome-mediated gene transfer and expression by transferrin. Hum. Gene Ther., 7, 275-282.
6. Curiel D.T. (1994) Gene delivery via the receptor-mediated pathway. In Wolff, J.A. (ed), Gene Therapeutics: Methods and Applications of Direct Gene Transfer (Boston: Birkhäuser), pp. 99-117.
7. Düzgünes N. and Feigner P.L. (1993) Intracellular delivery of nucleic acids and transcription factors by cationic liposomes. Meth. Enzymol., 221, 303-306.
8. Farhood H., Gao X., Son K., Yang Y.-Y., Lazo J.S., Huang L., Barsoum J., Bottega R. and Epand R.M. (1994) Cationic liposomes for direct gene transfer in therapy of cancer and other diseases. Ann. NY Acad. Sci., 716, 23-35.
9. Farhood H., Serbina N. and Hyang L. (1995) The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim. Biophys. Acta, 1235, 289-295.
10. Feigner P.L., Gadek T.R., Holm M., Roman R., Chan H.W., Wenz M., Northrop J.P., Ringold G.M. and Danielsen M. (1987) Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc. Natl. Acad. Sci. USA, 84, 7413-7417.
11. Feigner P.L., Tsai Y.J., Sukhu L., Wheeler C.J., Manthorpe M., Marshall J. and Cheng S.H. (1995) Improved cationic lipid formulations for in vivo gene therapy. Ann. N.Y. Acad. Sci., 772, 126-139.
12. Fields R.D. and Lancaster M.V. (1993) Dual-attribute continuous monitoring of cell proliferation/cytotoxicity. Am. Biotechnol. Lab., 11, 48-50.
13. Fisher A.C., Collatti E., Ratner L., Gallo R.C. and Wong-Staal F. (1985) A molecular clone of HTLV-III with biological activity. Nature, 316, 262-265.
14. Friend D.S., Papahadjopoulos D. and Debs R.J. (1996) Endocytosis and intracellular processing accompanying transfection mediated by cationic liposomes Biochim. Biophys. Acta, 1278, 41-50.
15. Good P.D., Krikos A.J., Li S.X.L., Bertrand E., Lee M.S., Giver L., Ellington A., Zaia J.A., Rossi J.J. and Engelke D.R. (1997) Expression of small, therapeutic RNAs in human cell nuclei. Gene Ther., 4, 45-54.
16. Harrison G.S., Long C.J., Maxwell F., Glode M. and Maxwell I.H. (1992) Inhibition of HIV production in cells containing an integrated, HIV-regulated diphtheria toxin A chain gene. AIDS Res. Hum. Retroviruses, 8, 39-45.
17. Heaphy S., Finch J.T., Gait M.J., Karn J. and Singh M. (1991) Human immunodeficiency virus type 1 regulator of virion expression, rev, forms nucleoprotein filaments after binding to a purine-rich "bubble" located within the rev-responsive region of viral mRNAs. Proc. Natl. Acad. Sci. USA, 88, 7366-7370.
18. Hug P. and Sleight R.G. (1991) Liposomes for the transformation of eukaryotic cells. Biochim. Biophys. Acta, 1097, 1-17.
19. Kjems J., Calnan B.J., Frankel A.D. and Sharp P.A. (1992) Specific-binding of a basic peptide from HIV-1 Rev. EMBO J., II, 1119-1129.
20. Konopka K., Davis B.R., Larsen C.E., Alford D.R., Debs R.J. and Düzgünes N. (1990) Liposomes modulate human immunodeficiency virus infectivity. J. Gen. Virol., 71, 2899-2907.
21. Konopka K., Pretzer E., Feigner P.L. and Düzgünes N. (1996) Human immunodeficiency virus type-1 (HIV-1) infection increases the sensitivity of macrophages and THP-1 cells to cytotoxicity by cationic liposomes. Biochim. Biophys. Acta, 1312, 186-196.
22. Konopka K., Harrison G.S., Felgner P.L. and Düzgünes N. (1997) Cationic liposome-mediated expression of HIV-regulated luciferase and diphtheria toxin A genes in HeLa cells infected with or expressing HIV. Biochim. Biophys. Acta, 1356, 185-197.
23. Lee S.-W., Gallardo H.F., Gilboa E. and Smith C. (1994) Inhibition of human immunodeficiency virus type 1 in human T cells by a potent Rev response element decoy comprised of the 13-nucleotide minimal Rev-binding domain. J. Virol., 68, 8254-8264.
24. Lee T.C., Sullenger B.A., Gallardo H.F., Ungers G.E. and Gilboa E. (1992) Overexpression of RRE-derived sequences inhibits HIV-1 replication in CEM cells. New Biol., 4, 55-74.
25. Legendre J.Y. and Szoka F.C. (1992) Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: comparison with cationic liposomes. Pharm. Res., 9, 1235-1242.
26. Liu Y., Liggitt D., Zhong W., Tu G., Gaensler K. and Debs R. (1995) Cationic liposome-mediated intravenous gene delivery. J. Biol. Chem., 270, 24864-24870.
27. Nabel E.G., Gordon D., Yang Z.-Y., Xu L., San H., Plautz G.E. , Wu B.-Y., Gao X., Huang L. and Nabel G.J. (1992a) Gene transfer in vivo with DNA-liposome complexes: Lack of autoimmunity and gonadal localization. Hum. Gene Ther., 3, 649-656.
28. Nabel G.J., Chang A., Nabel E.G., Plautz G., Fox B.A., Huang L. and Shu S. (1992b) Clinical protocol: Immunotherapy of malignancy by in vivo gene transfer into tumors. Hum. Gene Ther., 3, 399-410.
29. Philip R., Liggitt D., Philip M., Dazin P. and Debs R. (1993) In vivo gene delivery. Efficient transfection of T lymphocytes in adult mice. J. Biol. Chem., 268, 16087-6090.
30. Pinnaduwage P., Schmitt L. and Huang L. (1989) Use of quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells. Biochim. Biophys. Acta, 985, 33-37.
31. Remy J.-S., Kichler A., Mordvinov V., Schuber F. and Behr J.-P. (1995) Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: A stage toward artificial viruses. Proc. Natl. Acad. Sci. USA, 92, 1744-1748.
32. Rossi J.J., Elkins D., Zaia J.A. and Sullivan S. (1992) Ribozymes as anti-HIV-1 therapeutic agents: principles, applications, and problems. AIDS Res. Hum. Retroviruses, 8, 183-189.
33. Rossi J.J. and Sarver N. (1992) Catalytic antisense RNA (ribozymes): their potential and use as anti-HIV-1 therapeutic agents. Adv. Exp. Med. Biol., 312, 95-109.
34. Sarver N., Cantin E.M., Chang P.S., Zaia J.A., Ladne P.A., Stephens D.A. and Rossi J.J. (1990) Ribozymes as potential anti-HIV-1 therapeutic agents. Science, 247, 1222-1225.
35. Singhal A. and Huang L. (1994) Gene transfer in mammalian cells using liposomes as carriers. In Wolff, J.A. (ed). Gene Therapeutics: Methods and Applications of Direct Gene Transfer (Boston: Birkhäuser), pp. 118-142.
36. Stewart M.J., Plautz G.E., Del Buono L., Yang Z.Y., Xu L., Gao X., Huang L., Nabel E.G. and Nabel G.J. (1992) Gene transfer in vivo with DNA-liposome complexes: safety and acute toxicity in mice. Hum. Gene Ther., 3, 267-275.
37. Stribling R., Brunette E., Liggitt D., Gaensler K. and Debs R. (1992) Aerosol gene delivery in vivo. Proc. Natl. Acad. Sci. USA, 89, 11277-11281.
38. Thierry A.R., Lunardi-Iskandar Y., Bryant J.L., Rabinovich P., Gallo R.C. and Mahan L.C. (1995) Systemic gene therapy: biodistribution and long-term expression of a transgene in mice. Proc. Natl. Acad. Sci. USA, 92, 9742-9746.
39. Treco D.A. and Selden R.F. (1995) Non-viral gene therapy. Mol. Med. Today, 1, 314-321.
40. Tuerk C. and MacDougal-Waugh S. (1993) In vitro evolution of functional nucleic acids: high-affinity RNA ligands of HIV-1 proteins. Gene 137, 33-39.
41. Wagner E., Zenke M., Cotten M., Beug H. and Birnstiel M.L. (1990) Transferrin-polycation conjugates as carriers for DNA uptake into. cells. Proc. Natl. Acad Sci. USA, 87, 3410-3414.
42. Wrohel I. and Collins D. (1995) Fusion of cationic liposomes with mammalian cells occurs after endocytosis. Biochim. Biophys. Acta, 1235, 296-304.
43. Xu Y. and Szoka F.C.Jr. (1996) Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry, 35, 5616-5623.
44. Yamada O., Kraus G., Luznik L., Yu M. and Wong-Staal F. (1996) A chimeric human immunodeficiency virus type 1 (HIV-1) minimal Rev response element-ribozyme molecule exhibits dual antiviral function and inhibits cell-cell transmission of HIV-1. J. Virol., 70, 1596-1601.
45. Yu M., Poeschla E. and Wong-Staal F. (1994) Progress towards gene therapy for HIV infection. Gene Ther., 1, 13-26.
46. Zabner J., Fasbender A.J., Moninger T., Poellinger K.A. and Welsch M.J. (1995) Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem., 270, 18997-19007.
47. Zenke M., Steinlein P., Wagner E., Cotten M., Beug H. and Birnstiel M.L. (1990) Receptor-mediated endocytosis of transferrin-polycation conjugates: an efficient way to introduce DNA into hematopoietic cells. Proc. Natl. Acad. Sci. USA, 87, 3655-3659.