Skip to main content
SpringerLink
Log in

Functional Amphiphiles for Gene Delivery

  • Technical Feature
  • Published:
MRS Bulletin Aims and scope Submit manuscript

Abstract

The design of safe and efficient gene transfer vectors remains one of the key challenges in gene therapy. Despite their remarkable transfection efficiency, viral vectors suffer from known safety issues. Consequently, significant research activity has been undertaken to develop nonviral approaches to gene transfer during the last decade. Numerous academic and industrial research groups are investigating synthetic cationic vectors, such as cationic amphiphiles, with the objective of increasing the gene transfection activity. Within this area, the development of functional synthetic vectors that respond to local environmental effects have met with success. These synthetic vectors are based on mechanistic principles and represent a significant departure from earlier systems. Many of these systems for gene delivery in vitro and in vivo are discussed in this article.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. R. Weiss and D. Neldso, “Teen Dies Undergoing Gene Therapy,” The Washington Post, Sept. 29, 1999.

  2. P.L. Felgner, T.R. Gadek, M. Holm, R. Roman, H.W. Chan, M. Wenz, J.P. Northrop, G.M. Ringold, and M. Danielsen, Proc. Natl. Acad. Sci. USA 84 (1987) p. 7413.

    Article  CAS  Google Scholar 

  3. W.T. Godbey and A.G. Mikos, J. Controlled Release 72 (2001) p. 115.

    Article  CAS  Google Scholar 

  4. P.L. Felgner, T.R. Gadek, M. Holm, R. Roman, H.W. Chan, M. Wenz, J.P. Northrop, X. Gao, and L. Huang. Gene Ther. 2 (1995) p. 710.

    Google Scholar 

  5. A.P. Rolland, Crit. Rev. Ther. Drug Carrier Syst. 15 (1998) p. 143.

    Article  CAS  Google Scholar 

  6. J.Y. Legendre and F.C. Szoka, J. Pharm. Res. 9 (1992) p. 1235.

    CAS  Google Scholar 

  7. A.D. Miller, Angew. Chem. Int. Ed. 37 (1998) p. 1768.

    Article  Google Scholar 

  8. P.L. Felgner and G. Rhodes, Nature 349 (1991) p. 351; J. Remy, C. Sirlin, P. Vierling, and J.P. Behr, Bioconjugate Chem. 5 (1994) p. 647; N. Zhu, D. Liggott, Y. Liu, and R. Debs, Science 261 (1993) p. 209; H.M. Deshmukh and L. Huang, New J. Chem. 21 (1997) p. 113; J.P. Behr, B. Demeneix, J.P. Loeffler, and J. Perez-Mutul, Proc. Nat. Acad. Sci. USA 86 (1989) p. 6982; Y. Rui, S. Wang, P.S. Low, and D.H. Thompson, J. Am. Chem. Soc. 120 (1998) p. 11213; U. Schulze, H. Schmidt, and C.R. Safinya, Bioconjugate Chem. 10 (1999) p. 548; J.P. Vigneron, N. Oudrhiri, M. Fauquet, L. Vergely, J.C. Bradley, M. Basseville, P. Lehn, J.M. Lehn, and J.P. Vigneron, Proc. Nat. Acad. Sci. USA 93 (1996) p. 9682; R.C. MacDonald, G.W. Ashley, M.M. Shida, V.A. Rakhmanova, Y.S. Tarahovsky, D.P. Pantazatos, M.T. Kennedy, E.V. Pozharski, K.A. Baker, R.D. Jones, H.S. Rosenzweig, K.L. Choi, R. Qiu, and T.J. McIntosh, Biophys. J. 77 (5) (1999) p. 2612.

    Article  CAS  Google Scholar 

  9. R. Leventis, J.R. Silvius, Biochim. Biophys. Acta 1023 (1990) p. 124.

    Article  CAS  Google Scholar 

  10. X. Gao and L. Huang, Biochem. Biophys. Res. Commun. 179 (1991) p. 280.

    Article  CAS  Google Scholar 

  11. J.K. Rose, L. Buonocore, and M.A. Whitt, BioTechniques 10 (1991) p. 520.

    CAS  Google Scholar 

  12. I. Solodin, C.S. Brown, M.S. Bruno, C.Y. Chow, E.H. Jang, R.J. Debs, and T.D. Heath, Biochemistry 34 (1995) p. 13537.

    Article  CAS  Google Scholar 

  13. C. Boulanger, C. Di Giorgio, J. Gaucheron, and P. Vierling, Bioconjugate Chem. 15 (2004) p. 901 and references therein.

    Article  CAS  Google Scholar 

  14. J.S. Choi, D.K. Joo, C.H. Kim, K. Kim, and J.S. Park, J. Am. Chem. Soc. 122 (2000) p. 474; M.X. Tang, C.T. Redemann, and F.C. Szoka, Bioconjugate Chem. 7 (1996) p. 703; J.F. Kukowska-Latallo, A.U. Bielinska, J. Johnson, R. Spindler, D.A. Tomalia, and J.R. Baker Jr., Proc. Nat. Acad. Sci. USA 93 (1996) p. 4897; D. Luo, K. Haverstick, N. Belcheva, E. Han, and W.M. Saltzman, Macromol. 35 (2002) p. 3456.

    Article  CAS  Google Scholar 

  15. A.S. Hoffman, P.S. Stayton, O. Press, N. Murthy, C.A. Lackey, C. Cheung, F. Black, J. Campbell, N. Fausto, T.R. Kyriakides, and P. Bornstein, Polym. Adv. Technol. 13 (2002) p. 992.

    Article  CAS  Google Scholar 

  16. F. Barthel, J.S. Remy, J.P. Loeffler, and J.P. Behr, DNA Cell Biol. 12 (1993) p. 553.

    Article  CAS  Google Scholar 

  17. G. Ronsin, C. Perrin, P. Guédat, A. Kremer, P. Camilleri, and A.J. Kirby, Chem. Commun. 21 (2001) p. 2234.

    Article  CAS  Google Scholar 

  18. F. Shi and D. Hoekstra, J. Controlled Release 97 (2) (2004) p. 189.

    Article  CAS  Google Scholar 

  19. D.R. Sørensen, M. Leirdal, and M. Sioud, J. Mol. Biol. 327 (2003) p. 761; M.T. McManus, B.B. Haines, C.P. Dillon, C.E. Whitehurst, L. van Parijs, J. Chen, and P.A. Sharp, J. Immunol. 169 (2002) p. 5754.

    Article  CAS  Google Scholar 

  20. P. Pires, S. Simoes, S. Nir, R. Gaspar, N. Duzgunes, and M.C. Pedroso de Lima, Biochim. Biophys. Acta 1418 (1999) p. 71; J. Zabner, A.J. Fasbender, T. Moninger, K.A. Poellinger, and M.J. Welsh, J. Biol. Chem. 270 (1995) p. 18997; Y. Xu and F.C. Szoka Jr., Biochemistry 35 (1996) p. 5616; D.S. Friend, D. Papahadjopoulos, and R.J. Debs, Biochim. Biophys. Acta 1278 (1996) p. 41.

    Article  CAS  Google Scholar 

  21. S. Simoes, V. Slepushkin, R. Gaspar, M.C. Pedroso de Lima, and N. Duzgunes, Gene Ther. 6 (1999) p. 1798.

    Article  CAS  Google Scholar 

  22. S. Mukherjee, R.N. Ghosh, and F.R. Maxfield, Physiol. Rev. 77 (1997) p. 759.

    Article  CAS  Google Scholar 

  23. A.D. Miller, Angew. Chem. Int. Ed. 37 (1998) p. 1768.

    Article  Google Scholar 

  24. I. Wrobel and D. Collins, Biochim. Biophys. Acta 1235 (1995) p. 296.

    Article  Google Scholar 

  25. M.B. Bally, P. Harvie, F.M.P. Wong, S. Kong, E.K. Wasan, and D.L. Reimer, Adv. Drug Deliv. Rev. 38 (1999) p. 291; P.W. Cheng, Hum. Gene Ther. 7 (1996) p. 275; T. Girao, S. Simoes, P. Pires, S. Nir, and M.C. Pedroso de Lima, Biochim. Biophys. Acta 1510 (2001) p. 136.

    Article  CAS  Google Scholar 

  26. Y. Xu and F.C. Szoka Jr., Biochemistry 35 (1996) p. 5616.

    Article  CAS  Google Scholar 

  27. R.L. Page, S.P. Butler, A. Subramanian, F.C. Gwazdauskas, J.L. Johnson, and W.H. Velander, Transgenic Res. 4 (1995) p. 353.

    Article  CAS  Google Scholar 

  28. S. Brunner, T. Sauer, S. Carotta, M. Cotten, M. Saltik, and E. Wagner, Gene Ther. 7 (2000) p. 401; I. Mortimer, P. Tam, I. MacLachlan, R.W. Graham, E.G. Saravolac, and P.B. Joshi, Gene Ther. 6 (1999) p. 403; M. Wilke, E. Fortunati, M. Van den Broek, A.T. Hoogeveen, and B.J. Scholte, Gene Ther. 3 (1996) p. 1133.

    Article  CAS  Google Scholar 

  29. B. Talcott and M.S. Moore, Trends Cell Biology 9 (1999) p. 312.

    Article  CAS  Google Scholar 

  30. D. Lechardeur, A.S. Verkman, and G.L. Lukacs, Adv. Drug Deliv. Rev. 57 (2005) p. 755.

    Article  CAS  Google Scholar 

  31. G.D. Schmidt-Wolf and I.G.H. Schmidt-Wolf, Trends in Molecular Medicine 9 (2) (2003) p. 67.

    Article  CAS  Google Scholar 

  32. G.A. Brazeau, S. Attia, S. Poxon, and J.A. Hughes, Pharm. Res. 15 (1998) p. 680; P.R. Dash, M.L. Read, L.B. Barret, M.A. Wolfert, and L.W. Seymour, Gene Ther. 6 (1999) p. 643.

    Article  CAS  Google Scholar 

  33. M.C. Pedroso de Lima, S. Simoes, P. Pires, H. Faneca, and N. Duzgunes, Adv. Drug Deliv. Rev. 47 (2001) p. 277.

    Article  CAS  Google Scholar 

  34. B. Martin, M. Sainlos, A. Aissaoui, N. Oudrhiri, M. Hauchecorne, J.P. Vigneron, J.M. Lehn, and P. Lehn, Curr. Pharm. Des. 11 (3) (2005) p. 375.

    Article  CAS  Google Scholar 

  35. A. Gijsens, A. Derycke, L. Missiaen, D. De Vos, J. Huwyler, A. Eberle, and P. de Witte, Int. J. Cancer 101 (2002) p. 78; E. Bohl Kullberg, N. Bergstrand, J. Carlsson, K. Edwards, M. Johnsson, S. Sjöberg, and L. Gedda, Bioconjugate Chem. 13 (2002) p. 737; H.E. Hofland, C. Masson, S. Iginla, I. Osetinsky, J.A. Reddy, C.P. Leamon, D. Scherman, M. Bessodes, and P. Wils, Mol. Ther. 5 (2002) p. 739.

    Article  CAS  Google Scholar 

  36. J.A. Reddy, C. Abburi, H. Hofland, S.J. Howard, I. Vlahov, P. Wils, C.P. Leamon, Gene Ther. 9 (2002) p. 1542.

    Article  CAS  Google Scholar 

  37. G. Thurston, J.W. McLean, M. Rizen, P. Baluk, A. Haskell, T.J. Murphy, D. Hanahan, and D.M. McDonald, J. Clin. Invest. 101 (1998) p. 1401.

    Article  CAS  Google Scholar 

  38. R. Kunsfeld, G. Wickenhauser, U. Michaelis, M. Teifel, W. Umek, K. Naujoks, K. Wolff, and P. Petzelbauer, J. Invest. Dermatol. 120 (2003) p. 476; A. Papyan, A.M. Werner, I. Ischenko, M. Yezhelyev, M. Teifel, U. Michaelis, and C.J. Bruns, Proc. Amer. Assoc. Cancer Res. 45 (2004) p. 4104; H. Haas, B. Schulze, A. Werner, U. Michaelis, B. Sauer, and M. Teifel, Proc. Amer. Assoc. Cancer Res. 44 (2003) p. 1793.

    Article  Google Scholar 

  39. M. Murata, Y. Sugahara, S. Takahashi, and S. Ohnishi, J. Biochem. 102 (1987) p. 957.

    Article  CAS  Google Scholar 

  40. C. Plank, B. Oberhauser, K. Mechtler, C. Koch, and E. Wagner, J. Biol. Chem. 269 (1994) p. 12918.

    Article  CAS  Google Scholar 

  41. C. Pichon, I. Freulon, P. Midoux, R. Mayer, M. Monsigny, and A.-C. Roche, Antisense Nucleic Acid Drug Dev. 7 (1997) p. 335.

    Article  CAS  Google Scholar 

  42. A. Noguchi, T. Furuno, C. Kawaura, and M. Nakanishi, FEBS Lett. 433 (1998) p. 169.

    Article  CAS  Google Scholar 

  43. P. Harvie, F.M.P. Wong, and M.B. Bally, Biophys. J. 75 (1998) p. 1040.

    Article  CAS  Google Scholar 

  44. S. Mukherjee, R.N. Ghosh, and F.R. Maxfield, Physiol. Rev. 77 (1997) p. 759.

    Article  CAS  Google Scholar 

  45. J.A. Boomer, D.H. Thompson, and S.M. Sullivan, Pharm. Res. 19 (2002) p. 1292; J. Zhu, R.J. Munn, and M.H. Nantz, J. Am. Chem. Soc. 122 (2000) p. 2645; A. Aissaoui, B. Martin, E. Kan, N. Oudrhiri, M. Hauchecorne, J.-P. Vigneron, J.-M. Lehn, and P. Lehn, J. Med. Chem. 47 (2004) p. 5210; D. Luton, N. Oudrhiri, P. de Lagausie, A. Aissaoui, M. Hauchecorne, S. Julia, J.F. Ouru, Y. Aigrain, M. Peuchmaur, J.P. Vigneron, J.M. Lehn, and P. Lehn, J. Gene Med. 6 (2004) p. 328; T.G. Kim, S.Y. Kang, J.H. Kang, M.Y. Cho, J.I. Kim, S.H. Kim, and J.S. Kim, Bioconjugate Chem. 15 (2004) p. 326; R.J. Cristiano and D.T. Curiel, Cancer Gene Ther. 3 (1996) p. 49; Q. Leng and A.J. Mixson, Nucleic Acids Res. 33 (4) (2005) p. 40; D. Putnam, A.N. Zelikin, V.A. Izumrudov, and R. Langer, Biomaterials 24 (2003) p. 4425; T.G. Kim, S.Y. Kang, J.H. Kang, M.Y. Cho, J.I. Kim, S.H. Kim, and J.S. Kim, Bioconjugate Chem. 15 (2) (2004) p. 326; V.V. Kumar, C. Pichon, M. Refregiers, B. Guerin, P. Midoux, and A. Chaudhuri, Gene Ther. 10 (2003) p.1206.

    Article  CAS  Google Scholar 

  46. G. Wenjin, M.A. Gosselin, and R.J. Lee, J. Controlled Release 83 (2002) p. 121; S. Simões, J.N. Moreira, C. Fonseca, N. Düzgüne, and M.C. Pedroso de Lima, Adv. Drug Del. Rev. 56 (2004) p. 947; R.A. Jones, C.Y. Cheung, F.E. Black, J.K. Zia, P.S. Stayton, A.S. Hoffman, M.R. Wilson, Biochem. J. 372 (2003) p. 65; P.S. Stayton, A.S. Hoffman, N. Murthy, C. Lackey, C. Cheung, P. Tan, L.A. Klumb, A. Chilkoti, F.S. Wilbur, and O.W. Press, J. Controlled Release 65 (2000) p. 203; J. Zhu, R.J. Munn, and M.H. Nantz, J. Am. Chem. Soc. 122 (2000) p. 2645.

    Article  Google Scholar 

  47. G. Byk, B. Wetzer, M. Frederic, C. Dubertret, B. Pitard, G. Jaslin, and D. Scherman, J. Med. Chem. 43 (2000) p. 4377; M. Balakirev, G. Schoehn, and J. Chroboczek, Chem. Bio. 7 (10) 2000 p. 813; R.C. Carlisle, T. Etrych, S.S. Briggs, J.A. Preece, K. Ulbrich, and L.W. Seymour, J. Gene Med. 6 (2004) p. 337.

    Article  CAS  Google Scholar 

  48. B. Wetzer, G. Byk, M. Frederic, M. Airiau, F. Blanche, B. Pitard, and D. Scherman, Biochemical J. 356 (2001) p. 747.

    Article  CAS  Google Scholar 

  49. C. McGregor, C. Perrin, M. Monck, P. Camilleri, and A.J. Kirby, J. Am. Chem. Soc. 123 (2001) p. 6215; F. Tang and J.A. Hughes, Biochem. Biophys. Res. Commun. 242 (1998) p. 141; G. Byk, B. Wetzer, M. Frederic, C. Dubertret, B. Pitard, G. Jaslin, and D. Scherman, J. Med. Chem. 43 (2000) p. 4377.

    Article  CAS  Google Scholar 

  50. D. Lleres, J.P. Clamme, E. Dauty, T. Blessing, G. Krishnamoorthy, G. Duportail, and Y. Mely, Langmuir 18 (2002) p. 10340.

    Article  CAS  Google Scholar 

  51. Y. Mély, D. Llères, J.P. Clamme, E. Dauty, G. Krishnamoorthy, and G. Duportail, presented at DNA Supramolecular Assemblies Workshop, Avignon, France, May 5–6, 2004.

  52. J.P. Behr, B. Demeneix, J.P. Loeffler, and J. Perez-Mutul, Proc. Natl. Acad. Sci. USA 86 (1989) p. 6982.

    Article  CAS  Google Scholar 

  53. B. Schwartz, C. Benoist, B. Abdallah, D. Scherman, J.P. Behr, and B.A. Demeneix. Hum. Gene Ther. 6 (1995) p. 1515.

    Article  CAS  Google Scholar 

  54. J. Lin, N.L. Slack, A. Ahmad, C.X. George, C.C.R. Safinya, and C.E. Samuel, Biophys. J. 84 (2003) p. 3307.

    Article  CAS  Google Scholar 

  55. J. Zabner, A.J. Fasbender, T. Moninger, K.A. Poellinger, and M.J. Welsh. J. Biol. Chem. 270 (1995) p. 18997.

    Article  CAS  Google Scholar 

  56. C.A.H. Prata, Y. Zhao, P. Barthélémy, Y. Li, D. Luo, J.T. McIntosh, S.J. Lee, and M.W. Grinstaff, J. Am. Chem. Soc. 126 (2004) p. 12196.

    Article  CAS  Google Scholar 

  57. C.A.H. Prata, Y. Zhao, P. Barthélémy, Y. Li, D. Luo, T.J. McIntosh, S.J. Lee, and M.W. Grinstaff, presented at DNA Supramolecular Assemblies Workshop, Avignon, France, May 5–6, 2004.

  58. P. Barthélémy, C.A.H. Prata, S.F. Filocamo, C.E. Immoos, B.W. Maynor, S.J. Lee, and M.W. Grinstaff, Chem. Commun. 14 (10) (2005) p. 1261.

    Article  CAS  Google Scholar 

  59. N. Campins, L. Moreau, M.W. Grinstaff, and P. Barthélémy, presented at DNA Supramolecular Assemblies Workshop, Avignon, France, May 5–6, 2004.

  60. P. Chabaud, M. Camplo, D. Payet, G. Serin, L. Moreau, P. Barthélémy, and M.W. Grinstaff, Bioconjugate Chem. (2005) submitted; L. Moreau, P. Barthélémy, Y. Li, D. Luo, C.A.H. Prata, and M.W. Grinstaff, Mol. BioSystems (2005) DOI:10. 1039/b503302k.

  61. L. Moreau, P. Barthélémy, M. El Maataoui, and M.W. Grinstaff. J. Am. Chem. Soc. 126 (2004) p. 7533.

    Article  CAS  Google Scholar 

  62. L. Moreau, M.W. Grinstaff, and P. Barthélémy. Tetrahedron Lett. 46 (2005) p. 1593.

    Article  CAS  Google Scholar 

  63. A.E. Regelin, E. Fernholz, H.F. Krug, and U. Massing, J. Biomol. Screen. 6 (4) (2001) p. 245.

    Article  CAS  Google Scholar 

  64. S. Armknecht, M. Boutros, A. Kiger, K. Nybakken, B. Mathey-Prevot, and N. Perrimon, Methods Enzymol. 392 (2005) p. 55.

    Article  CAS  Google Scholar 

Download references

Authors
  1. Philippe Barthélémy
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Michel Camplo
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Barthélémy, P., Camplo, M. Functional Amphiphiles for Gene Delivery. MRS Bulletin 30, 647–653 (2005). https://doi.org/10.1557/mrs2005.191

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1557/mrs2005.191

Keywords

Search

Navigation