Preclinical development and ocular biodistribution of gemini-DNA nanoparticles after intravitreal and topical administration: Towards non-invasive glaucoma gene therapy

Nanomedicine: Nanotechnology, Biology, and Medicine

Volumn 10, Issue 8, 2014, Pages 1637-1647

  Alqawlaq, Samih ^a   Sivak, Jeremy M ^c,d   Huzil, J Torin ^a   Ivanova, Marina V ^a   Flanagan, John G ^b,c,d   Beazely, Michael A ^a   Foldvari, Marianna ^a  

^a UNIVERSITY OF WATERLOO   (Canada)

^b UNIVERSITY OF WATERLOO   (Canada)

^c UNIVERSITY OF TORONTO   (Canada)

^d UNIVERSITY HEALTH NETWORK   (Canada)

Author keywords

Gene delivery; Glaucoma; Nanoparticles; Non viral; Ocular administration; Preclinical; Retina; Topical

Indexed keywords

BIOCOMPATIBILITY; DRUG THERAPY; EYE MOVEMENTS; GENE THERAPY; GENE TRANSFER; MAMMALS; NANOPARTICLES; PHOSPHOLIPIDS; SURFACE ACTIVE AGENTS;

GENE DELIVERY; GLAUCOMA; NONVIRAL; PRECLINICAL; RETINA; TOPICAL;

OPHTHALMOLOGY;

EYE DROPS; NANOPARTICLE; PHOSPHOLIPID; PLASMID DNA; SURFACTANT;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTERIOR EYE CHAMBER; ARTICLE; BIOCOMPATIBILITY; CELL VIABILITY; CONJUNCTIVA; CONTROLLED STUDY; CORNEA LIMBUS; DRUG BLOOD LEVEL; DRUG DISPOSITION; DRUG SCREENING; GLAUCOMA; IRIS; LACRIMAL FLUID; MALE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; RAT; RETINA GANGLION CELL; VITREOUS BODY; ANIMAL; C57BL MOUSE; CHEMISTRY; EYE; GENE THERAPY; GENE TRANSFER; METABOLISM; MOUSE; PROCEDURES; TOPICAL DRUG ADMINISTRATION;

ADMINISTRATION, TOPICAL; ANIMALS; EYE; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GLAUCOMA; MALE; MICE; MICE, INBRED C57BL; NANOPARTICLES;

EID: 84908576353     PISSN: 15499634     EISSN: 15499642     Source Type: Journal    
DOI: 10.1016/j.nano.2014.05.010     Document Type: Article

References (50)

1. Quigley H.A., Broman A.T. The number of people with glaucoma worldwide in 2010 and 2020. Br J Ophthalmol 2006, 90:262-267.
2. Sommer A., et al. Relationship between intraocular pressure and primary open angle glaucoma among white and black Americans. The Baltimore Eye Survey. Arch Ophthalmol 1991, 109:1090-1095.
3. Tielsch J.M., Katz J., Quigley H.A., Javitt J.C., Sommer A. Diabetes, intraocular pressure, and primary open-angle glaucoma in the Baltimore Eye Survey. Ophthalmology 1995, 102:48-53.
4. Yanagi M., et al. Vascular risk factors in glaucoma: a review. Clin Experiment Ophthalmol 2011, 39:252-258.
5. Sigal I.A., Flanagan J.G., Ethier C.R. Factors influencing optic nerve head biomechanics. Invest Ophthalmol Vis Sci 2005, 46:4189-4199.
6. Sigal I.A., Flanagan J.G., Tertinegg I., Ethier C.R. Finite element modeling of optic nerve head biomechanics. Invest Ophthalmol Vis Sci 2004, 45:4378-4387.
7. Demetriades A.M. Gene therapy for glaucoma. Curr Opin Ophthalmol 2011, 22:73-77.
8. Liu X., Rasmussen C.A., Gabelt B.T., Brandt C.R., Kaufman P.L. Gene therapy targeting glaucoma: where are we?. Surv Ophthalmol 2009, 54:472-486.
9. Del Amo E.M., Urtti A. Current and future ophthalmic drug delivery systems. A shift to the posterior segment. Drug Discov Today 2008, 13:135-143.
10. Mintzer M.A., Simanek E.E. Nonviral vectors for gene delivery. Chem Rev 2008, 109:259-302.
11. Puras G., et al. Low molecular weight oligochitosans for non-viral retinal gene therapy. Eur J Pharm Biopharm 2012, 10.1016/j.ejpb.2012.09.010.
12. Puras G., et al. Oligochitosan polyplexes as carriers for retinal gene delivery. Eur J Pharm Sci 2013, 48:323-331.
13. Morille M., Passirani C., Vonarbourg A., Clavreul A., Benoit J.P. Progress in developing cationic vectors for non-viral systemic gene therapy against cancer. Biomaterials 2008, 29:3477-3496.
14. De Laporte L., Cruz Rea J., Shea L.D. Design of modular non-viral gene therapy vectors. Biomaterials 2006, 27:947-954.
15. Donkuru M., et al. Advancing nonviral gene delivery: lipid- and surfactant-based nanoparticle design strategies. Nanomedicine 2010, 5:1103-1127.
16. Liaw J., Chang S.F., Hsiao F.C. In vivo gene delivery into ocular tissues by eye drops of poly(ethylene oxide)-poly(propylene oxide)-poly(ethylene oxide) (PEO-PPO-PEO) polymeric micelles. Gene Ther 2001, 8:999-1004.
17. Tong Y.C., et al. Eye drop delivery of nano-polymeric micelle formulated genes with cornea-specific promoters. J Gene Med 2007, 9:956-966.
18. Masuda I., Matsuo T., Yasuda T., Matsuo N. Gene transfer with liposomes to the intraocular tissues by different routes of administration. Invest Ophthalmol Vis Sci 1996, 37:1914-1920.
19. Matsuo T., Masuda I., Yasuda T., Matsuo N. Gene transfer to the retina of rat by liposome eye drops. Biochem Biophys Res Commun 1996, 219:947-950.
20. Johnson L.N., Cashman S.M., Kumar-Singh R. Cell-penetrating peptide for enhanced delivery of nucleic acids and drugs to ocular tissues including retina and cornea. Mol Ther 2008, 16:107-114.
21. Hait S.K., Moulik S.P. Gemini surfactants: a distinct class of self-assembling molecules. Curr Sci India 2002, 82:1101-1111.
22. Wettig S.D., Verrall R.E., Foldvari M. Gemini surfactants: a new family of building blocks for non-viral gene delivery systems. Curr Gene Ther 2008, 8:9-23.
23. Badea I., et al. In vivo cutaneous interferon-gamma gene delivery using novel dicationic (gemini) surfactant-plasmid complexes. J Gene Med 2005, 7:1200-1214.
24. Wettig S.D., Badea I., Donkuru M., Verrall R.E., Foldvari M. Structural and transfection properties of amine-substituted gemini surfactant-based nanoparticles. J Gene Med 2007, 9:649-658.
25. Donkuru M., Wettig S.D., Verrall R.E., Badea I., Foldvari M. Designing pH-sensitive gemini nanoparticles for non-viral gene delivery into keratinocytes. J Mater Chem 2012, 22:6232-6244.
26. Yang P., et al. Enhanced gene expression in epithelial cells transfected with amino acid-substituted gemini nanoparticles. Eur J Pharm Biopharm 2010, 75:311-320.
27. Badea I., Virtanen C., Verrall R.E., Rosenberg A., Foldvari M. Effect of topical interferon-gamma gene therapy using gemini nanoparticles on pathophysiological markers of cutaneous scleroderma in Tsk/+mice. Gene Ther 2012, 19:978-987.
28. Alqawlaq S., Huzil J.T., Ivanova M.V., Foldvari M. Challenges in neuroprotective nanomedicine development: progress towards noninvasive gene therapy of glaucoma. Nanomedicine (Lond) 2012, 7:1067-1083.
29. Pitkanen L., Ruponen M., Nieminen J., Urtti A. Vitreous is a barrier in nonviral gene transfer by cationic lipids and polymers. Pharm Res 2003, 20:576-583.
30. Singh J., Michel D., Chitanda J.M., Verrall R.E., Badea I. Evaluation of cellular uptake and intracellular trafficking as determining factors of gene expression for amino acid-substituted gemini surfactant-based DNA nanoparticles. J Nanobiotechnol 2012, 10:7.
31. Rejman J., Oberle V., Zuhorn I.S., Hoekstra D. Size-dependent internalization of particles via the pathways of clathrin- and caveolae-mediated endocytosis. Biochem J 2004, 377:159-169.
32. Orsi M., Essex J.W. Physical properties of mixed bilayers containing lamellar and nonlamellar lipids: insights from coarse-grain molecular dynamics simulations. Faraday Discuss 2013, 161:249-272.
33. Kirk G.L., Gruner S.M. Lyotropic effects of alkanes and headgroup composition on the lα-Hii lipid liquid crystal phase transition: hydrocarbon packing versus intrinsic curvature. J Phys Fr 1985, 46:761-769.
34. Marrink S.J., Mark A.E. Molecular view of hexagonal phase formation in phospholipid membranes. Biophys J 2004, 87:3894-3900.
35. Foldvari M., Badea I., Wettig S., Verrall R., Bagonluri M. Structural characterization of novel gemini non-viral DNA delivery systems for cutaneous gene therapy. J Exp Nanosci 2006, 1:165-176.
36. Pinnaduwage P., Schmitt L., Huang L. Use of a quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells. Biochim Biophys Acta 1989, 985:33-37.
37. Kawakami S., Yamashita F., Nishikawa M., Takakura Y., Hashida M. Asialoglycoprotein receptor-mediated gene transfer using novel galactosylated cationic liposomes. Biochem Biophys Res Commun 1998, 252:78-83.
38. Kearns M.D., Patel Y.N., Savva M. Physicochemical characteristics associated with transfection of cationic cholesterol-based gene delivery vectors in the presence of DOPE. Chem Phys Lipids 2010, 163:755-764.
39. Piest M., Engbersen J.F. Effects of charge density and hydrophobicity of poly(amido amine)s for non-viral gene delivery. J Control Release 2010, 148:83-90.
40. Lappalainen K., Jaaskelainen I., Syrjanen K., Urtti A., Syrjanen S. Comparison of cell proliferation and toxicity assays using two cationic liposomes. Pharm Res 1994, 11:1127-1131.
41. Aberle A.M., et al. A novel tetraester construct that reduces cationic lipid-associated cytotoxicity. Implications for the onset of cytotoxicity. Biochemistry 1998, 37:6533-6540.
42. Lv H., Zhang S., Wang B., Cui S., Yan J. Toxicity of cationic lipids and cationic polymers in gene delivery. J Control Release 2006, 114:100-109.
43. Kolle S.N., Kandarova H., Wareing B., van Ravenzwaay B., Landsiedel R. In-house validation of the EpiOcular(TM) eye irritation test and its combination with the bovine corneal opacity and permeability test for the assessment of ocular irritation. Altern Lab Anim 2011, 39:365-387.
44. Schaeffer H.E., Krohn D.L. Liposomes in topical drug delivery. Invest Ophthalmol Vis Sci 1982, 22:220-227.
45. Hamalainen K.M., Kananen K., Auriola S., Kontturi K., Urtti A. Characterization of paracellular and aqueous penetration routes in cornea, conjunctiva, and sclera. Invest Ophthalmol Vis Sci 1997, 38:627-634.
46. Wadhwa S., Paliwal R., Paliwal S.R., Vyas S.P. Nanocarriers in ocular drug delivery: an update review. Curr Pharm Des 2009, 15:2724-2750.
47. Peeters L., et al. Vitreous: a barrier to nonviral ocular gene therapy. Invest Ophthalmol Vis Sci 2005, 46:3553-3561.
48. Koo H., et al. The movement of self-assembled amphiphilic polymeric nanoparticles in the vitreous and retina after intravitreal injection. Biomaterials 2012, 33:3485-3493.
49. Urtti A. Challenges and obstacles of ocular pharmacokinetics and drug delivery. Adv Drug Deliv Rev 2006, 58:1131-1135.
50. Gaudana R., Ananthula H.K., Parenky A., Mitra A.K. Ocular drug delivery. AAPS J 2010, 12:348-360.