Wanted and unwanted properties of surface PEGylated nucleic acid nanoparticles in ocular gene transfer

Journal of Controlled Release

Volumn 122, Issue 3, 2007, Pages 226-235

  Sanders, Niek N ^a   Peeters, Liesbeth ^a   Lentacker, Ine ^a   Demeester, Joseph ^a   De Smedt, Stefaan C ^a  

^a GHENT UNIVERSITY   (Belgium)

Author keywords

Lipoplexes; Microbubble; Ocular gene therapy; Ultrasound; Vitreous

Indexed keywords

DISEASES; GENE TRANSFER; MOLECULAR WEIGHT; NANOPARTICLES; ULTRASONICS;

INTRAVITREAL INJECTION; LIPOPLEXES; MICROBUBBLES; OCULAR GENE THERAPY;

NUCLEIC ACIDS;

LIPOPLEX; MACROGOL; NANOPARTICLE; NUCLEIC ACID;

ANIMAL TISSUE; ARTICLE; CHEMICAL STRUCTURE; CONTROLLED STUDY; GENE DELIVERY SYSTEM; GENE TRANSFER; INTERNALIZATION; MICROBUBBLE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PIGMENT EPITHELIUM; PRIORITY JOURNAL; STRUCTURE ANALYSIS; SURFACE PROPERTY; VITREOUS BODY;

ANIMALS; CATTLE; DRUG CARRIERS; DRUG STABILITY; EPITHELIAL CELLS; EYE DISEASES; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LIPOSOMES; LUCIFERASES, FIREFLY; MICROBUBBLES; NANOPARTICLES; NUCLEIC ACIDS; PIGMENT EPITHELIUM OF EYE; POLYETHYLENE GLYCOLS; TRANSFECTION; ULTRASONICS; VITREOUS BODY;

EID: 34548623989     PISSN: 01683659     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jconrel.2007.05.004     Document Type: Article

References (38)

1. Acland G.M., Aguirre G.D., Ray J., Zhang Q., Aleman T.S., Cideciyan A.V., Pearce-Kelling S.E., Anand V., Zeng Y., Maguire A.M., Jacobson S.G., Hauswirth W.W., and Bennett J. Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet. 28 (2001) 92-95
2. Acland G.M., Aguirre G.D., Bennett J., Aleman T.S., Cideciyan A.V., Bennicelli J., Dejneka N.S., Pearce-Kelling S.E., Maguire A.M., Palczewski K., Hauswirth W.W., and Jacobson S.G. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol. Ther. 12 (2005) 1072-1082
3. Bainbridge J.W.B., Tan M.H., and Ali R.R. Gene therapy progress and prospects: the eye. Gene Ther. 13 (2006) 1191-1197
4. Andrieu-Soler C., Bejjani R.A., de Bizemont T., Normand N., BenEzra D., and Behar-Cohen F. Ocular gene therapy: a review of nonviral strategies. Mol. Vis. 12 (2006) 1334-1347
5. Bloquel C., Bourges J.L., Touchard E., Berdugo M., BenEzra D., and Behar-Cohen F. Non-viral ocular gene therapy: potential ocular therapeutic avenues. Adv. Drug Deliv. Rev. 58 (2006) 1224-1242
6. Duvvuri S., Majumdar S., and Mitra A.K. Drug delivery to the retina: challenges and opportunities. Expert. Opin. Biol. Ther. 3 (2003) 45-56
7. Sakamoto T., Ikeda Y., and Yonemitsu Y. Gene targeting to the retina. Adv. Drug Deliv. Rev. 52 (2001) 93-102
8. Scott J.E. The chemical morphology of the vitreous. Eye 6 (1992) 553-555
9. Bishop P. The biochemical structure of mammalian vitreous. Eye 10 (1996) 664-670
10. Ruponen M., Honkakoski P., Ronkko S., Pelkonen J., Tammi M., and Urtti A. Extracellular and intracellular barriers in non-viral gene delivery. J. Control. Release 93 (2003) 213-217
11. Ruponen M., Ronkko S., Honkakoski P., Pelkonen J., Tammi M., and Urtti A. Extracellular glycosaminoglycans modify cellular trafficking of lipoplexes and polyplexes. J. Biol. Chem. 276 (2001) 33875-33880
12. Pitkanen L., Ruponen M., Nieminen J., and Urtti A. Vitreous is a barrier in nonviral gene transfer by cationic lipids and polymers. Pharm. Res. 20 (2003) 576-583
13. Braeckmans K., Peeters L., Sanders N.N., De Smedt S.C., and Demeester J. Three-dimensional fluorescence recovery after photobleaching with the confocal scanning laser microscope. Biophys. J. 85 (2003) 2240-2252
14. Davis A.A., Bernstein P.S., Bok D., Turner J., Nachtigal M., and Hunt R.C. A human retinal pigment epithelial cell line that retains epithelial characteristics after prolonged culture. Invest. Ophthalmol. Vis. Sci. 36 (1995) 955-964
15. Quax P.H.A., Vanmuijen G.N.P., Weeningverhoeff E.J.D., Lund L.R., Dano K., Ruiter D.J., and Verheijen J.H. Metastatic behavior of human-melanoma cell-lines in nude-mice correlates with urokinase-type plasminogen-activator, its type-1 inhibitor, and urokinase-mediated matrix degradation. J. Cell Biol. 115 (1991) 191-199
16. Sanders N.N., Van Rompaey E., De Smedt S.C., and Demeester J. Structural alterations of gene complexes by cystic fibrosis sputum. Am. J. Respir. Crit. Care Med. 164 (2001) 486-493
17. Sanders N.N., De Smedt S.C., Cheng S.H., and Demeester J. Pegylated GL67 lipoplexes retain their gene transfection activity after exposure to components of CF mucus. Gene Ther. 9 (2002) 363-371
18. Peeters L., Sanders N.N., Braeckmans K., Boussery K., de Voorde J.V., De Smedt S.C., and Demeester J. Vitreous: a barrier to nonviral ocular gene therapy. Invest. Ophthalmol. Vis. Sci. 46 (2005) 3553-3561
19. Bradford M.M. A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal. Biochem. 72 (1976) 248-254
20. Li S., Tseng W.C., Stolz D.B., Wu S.P., Watkins S.C., and Huang L. Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection. Gene Ther. 6 (1999) 585-594
21. Zelphati O., Uyechi L.S., Barron L.G., and Szoka F.C. Effect of serum components on the physico-chemical properties of cationic lipid/oligonucleotide complexes and on their interactions with cells. Biochim. Biophys. Acta L, Lipids Lipid Metab. 1390 (1998) 119-133
22. Sanders N.N., De-Smedt S.C., Van-Rompaey E., Simoens P., De-Baets F., and Demeester J. Cystic fibrosis sputum - a barrier to the transport of nanospheres. Am. J. Respir. Crit. Care Med. 162 (2000) 1905-1911
23. Maurer N., Mori A., Palmer L., Monck M.A., Mok K.W.C., Mui B., Akhong Q.F., and Cullis P.R. Lipid-based systems for the intracellular delivery of genetic drugs. Mol. Membr. Biol. 16 (1999) 129-140
24. Meyer M., and Wagner E. pH-responsive shielding of non-viral gene vectors. Expert Opin. Drug Deliv. 3 (2006) 563-571
25. Hoekstra D., Rejman J., Wasungu L., Shi F., and Zuhorn I. Gene delivery by cationic lipids: in and out of an endosome. Biochem. Soc. Trans. 35 (2007) 68-71
26. Wheeler J.J., Palmer L., Ossanlou M., MacLachlan I., Graham R.W., Zhang Y.P., Hope M.J., Scherrer P., and Cullis P.R. Stabilized plasmid-lipid particles: construction and characterization. Gene Ther. 6 (1999) 271-281
27. Rejman J., Wagenaar A., Engberts J.B., and Hoekstra D. Characterization and transfection properties of lipoplexes stabilized with novel exchangeable polyethylene glycol-lipid conjugates. Biochim. Biophys. Acta 1660 (2004) 41-52
28. L. Peeters, N.N. Sanders, A. Jones, J. Demeester, S.C. De Smedt, Post-Pegylated lipoplexes are promising vehicles for gene delivery in Rpe cells, J. Control. Release (in press).
29. Unger E.C., Porter T., Culp W., Labell R., Matsunaga T., and Zutshi R. Therapeutic applications of lipid-coated microbubbles. Adv. Drug Deliv. Rev. 56 (2004) 1291-1314
30. Mehier-Humbert S., Bettinger T., Yan F., and Guy R.H. Plasma membrane poration induced by ultrasound exposure: implication for drug delivery. J. Control. Release 104 (2005) 213-222
31. I. Lentacker, S.C. De Smedt, J. Demeester, V. Van Marck, M. Bracke, N.N. Sanders, Lipoplex loaded microbubbles for gene delivery: a Trojan horse controlled by ultrasound, Adv. Funct. Mater. (in press).
32. Ogris M., Brunner S., Schuller S., Kircheis R., and Wagner E. PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther. 6 (1999) 595-605
33. Sanders N.N., De Smedt S.C., and Demeester J. Mobility and stability of gene complexes in biogels. J. Control. Release 87 (2003) 117-129
34. Tam P., Monck M., Lee D., Ludkovski O., Leng E.C., Clow K., Stark H., Scherrer P., Graham R.W., and Cullis P.R. Stabilized plasmid-lipid particles for systemic gene therapy. Gene Ther. 7 (2000) 1867-1874
35. Song L.Y., Ahkong Q.F., Rong Q., Wang Z., Ansell S., Hope M.J., and Mui B. Characterization of the inhibitory effect of PEG-lipid conjugates on the intracellular delivery of plasmid and antisense DNA mediated by cationic lipid liposomes. Biochim. Biophys. Acta, Biomembr. 1558 (2002) 1-13
36. Deshpande M.C., Davies M.C., Garnett M.C., Williams P.M., Armitage D., Bailey L., Vamvakaki M., Armes S.P., and Stolnik S. The effect of poly(ethylene glycol) molecular architecture on cellular interaction and uptake of DNA complexes. J. Control. Release 97 (2004) 143-156
37. Shi F., Wasungu L., Nomden A., Stuart M.C., Polushkin E., Engberts J.B., and Hoekstra D. Interference of poly(ethylene glycol)-lipid analogues with cationic-lipid-mediated delivery of oligonucleotides; role of lipid exchangeability and non-lamellar transitions. Biochem. J. 366 (2002) 333-341
38. Deshpande M.C., and Prausnitz M.R. Synergistic effect of ultrasound and PEI on DNA transfection in vitro. J. Control. Release 118 (2007) 126-135