Nanoparticle-based technologies for retinal gene therapy

European Journal of Pharmaceutics and Biopharmaceutics

Volumn 95, Issue , 2015, Pages 353-367

  Adijanto, Jeffrey ^a   Naash, Muna I ^a  

^a UNIVERSITY OF OKLAHOMA COLLEGE OF MEDICINE   (United States)

Author keywords

CK30; Nanoparticles; Non viral retinal gene therapy; PLGA; Polylysine; Retinal disease; Vector engineering

Indexed keywords

ALBUMIN NANOPARTICLE; DENDRIMER; GELATIN; GELATIN NANOPARTICLE; GOLD NANOPARTICLE; LIPOSOME; METAL NANOPARTICLE; NANOPARTICLE; POLYGLACTIN; POLYLACTIC ACID; POLYMER; POLYSACCHARIDE NANOPARTICLE; SOLID LIPID NANOPARTICLE; UNCLASSIFIED DRUG; DNA;

ARTICLE; CELLS BY BODY ANATOMY; ENDOSOME; EPIGENETICS; GENE EXPRESSION; GENE SILENCING; GENE THERAPY; HUMAN; IN VIVO GENE TRANSFER; NONHUMAN; NONVIRAL GENE THERAPY; PIGMENT EPITHELIUM; PROMOTER REGION; RETINA; RETINA CELL; RETINA DISEASE; TRANSGENE; ANIMAL; CYTOLOGY; DISEASE MODEL; GENE EXPRESSION REGULATION; GENE TRANSFER; GENETICS; METABOLISM; MOUSE; NANOTECHNOLOGY; PATHOPHYSIOLOGY; PROCEDURES; RETINAL DISEASES;

ANIMALS; DISEASE MODELS, ANIMAL; DNA; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HUMANS; MICE; NANOPARTICLES; NANOTECHNOLOGY; RETINA; RETINAL DISEASES;

EID: 84928496635     PISSN: 09396411     EISSN: 18733441     Source Type: Journal    
DOI: 10.1016/j.ejpb.2014.12.028     Document Type: Article

References (237)

1. Z. Han, S.M. Conley, R. Makkia, J. Guo, M.J. Cooper, and M.I. Naash Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery PLoS One 7 2012 e52189
2. Z. Han, S.M. Conley, and M.I. Naash AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects Invest. Ophthalmol. Vis. Sci. 52 2011 3051 3059
3. O. Simo-Servat, C. Hernandez, and R. Simo Genetics in diabetic retinopathy: current concepts and new insights Curr. Genomics 14 2013 289 299
4. S. Haddad, C.A. Chen, S.L. Santangelo, and J.M. Seddon The genetics of age-related macular degeneration: a review of progress to date Surv. Ophthalmol. 51 2006 316 363
5. A. Rattner, H. Sun, and J. Nathans Molecular genetics of human retinal disease Annu. Rev. Genet. 33 1999 89 131
6. S. Ferrari, E. Di Iorio, V. Barbaro, D. Ponzin, F.S. Sorrentino, and F. Parmeggiani Retinitis pigmentosa: genes and disease mechanisms Curr. Genomics 12 2011 238 249
7. B.J. Klevering, A. Maugeri, A. Wagner, S.L. Go, C. Vink, F.P. Cremers, and C.B. Hoyng Three families displaying the combination of Stargardt's disease with cone-rod dystrophy or retinitis pigmentosa Ophthalmology 111 2004 546 553
8. T. Fukui, S. Yamamoto, K. Nakano, M. Tsujikawa, H. Morimura, K. Nishida, N. Ohguro, T. Fujikado, M. Irifune, K. Kuniyoshi, A.A. Okada, A. Hirakata, Y. Miyake, and Y. Tano ABCA4 gene mutations in Japanese patients with Stargardt disease and retinitis pigmentosa Invest. Ophthalmol. Vis. Sci. 43 2002 2819 2824
9. H. Morimura, G.A. Fishman, S.A. Grover, A.B. Fulton, E.L. Berson, and T.P. Dryja Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosis Proc. Natl. Acad. Sci. USA 95 1998 3088 3093
10. T.B. Friedman, J.M. Schultz, Z.M. Ahmed, E.T. Tsilou, and C.C. Brewer Usher syndrome: hearing loss with vision loss Adv. Otorhinolaryngol. 70 2011 56 65
11. D. Cosgrove, and M. Zallocchi Usher protein functions in hair cells and photoreceptors Int. J. Biochem. Cell Biol. 46 2014 80 89
12. A.I. den Hollander, R. Roepman, R.K. Koenekoop, and F.P. Cremers Leber congenital amaurosis: genes, proteins and disease mechanisms Prog. Retin. Eye Res. 27 2008 391 419
13. N. Overlack, T. Goldmann, U. Wolfrum, and K. Nagel-Wolfrum Gene repair of an Usher syndrome causing mutation by zinc-finger nuclease mediated homologous recombination Invest. Ophthalmol. Vis. Sci. 53 2012 4140 4146
14. B.E. Low, M.P. Krebs, J.K. Joung, S.Q. Tsai, P.M. Nishina, and M.V. Wiles Correction of the Crb1rd8 allele and retinal phenotype in C57BL/6N mice via TALEN-mediated homology-directed repair Invest. Ophthalmol. Vis. Sci. 55 2014 387 395
15. N. Chadderton, S. Millington-Ward, A. Palfi, M. O'Reilly, G. Tuohy, M.M. Humphries, T. Li, P. Humphries, P.F. Kenna, and G.J. Farrar Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy Mol. Ther. 17 2009 593 599
16. S. Millington-Ward, N. Chadderton, M. O'Reilly, A. Palfi, T. Goldmann, C. Kilty, M. Humphries, U. Wolfrum, J. Bennett, P. Humphries, P.F. Kenna, and G.J. Farrar Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa Mol. Ther. 19 2011 642 649
17. J.W. Streilein Ocular immune privilege: the eye takes a dim but practical view of immunity and inflammation J. Leukoc. Biol. 74 2003 179 185
18. S. Kachi, Y. Oshima, N. Esumi, M. Kachi, B. Rogers, D.J. Zack, and P.A. Campochiaro Nonviral ocular gene transfer Gene Ther. 12 2005 843 851
19. R. Farjo, J. Skaggs, A.B. Quiambao, M.J. Cooper, and M.I. Naash Efficient non-viral ocular gene transfer with compacted DNA nanoparticles PLoS One 1 2006 e38
20. A.M. Maguire, F. Simonelli, E.A. Pierce, E.N. Pugh Jr., F. Mingozzi, J. Bennicelli, S. Banfi, K.A. Marshall, F. Testa, E.M. Surace, S. Rossi, A. Lyubarsky, V.R. Arruda, B. Konkle, E. Stone, J. Sun, J. Jacobs, L. Dell'Osso, R. Hertle, J.X. Ma, T.M. Redmond, X. Zhu, B. Hauck, O. Zelenaia, K.S. Shindler, M.G. Maguire, J.F. Wright, N.J. Volpe, J.W. McDonnell, A. Auricchio, K.A. High, and J. Bennett Safety and efficacy of gene transfer for Leber's congenital amaurosis N. Engl. J. Med. 358 2008 2240 2248
21. J.W. Bainbridge, A.J. Smith, S.S. Barker, S. Robbie, R. Henderson, K. Balaggan, A. Viswanathan, G.E. Holder, A. Stockman, N. Tyler, S. Petersen-Jones, S.S. Bhattacharya, A.J. Thrasher, F.W. Fitzke, B.J. Carter, G.S. Rubin, A.T. Moore, and R.R. Ali Effect of gene therapy on visual function in Leber's congenital amaurosis N. Engl. J. Med. 358 2008 2231 2239
22. A.V. Cideciyan, T.S. Aleman, S.L. Boye, S.B. Schwartz, S. Kaushal, A.J. Roman, J.J. Pang, A. Sumaroka, E.A. Windsor, J.M. Wilson, T.R. Flotte, G.A. Fishman, E. Heon, E.M. Stone, B.J. Byrne, S.G. Jacobson, and W.W. Hauswirth Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics Proc. Natl. Acad. Sci. USA 105 2008 15112 15117
23. A.V. Cideciyan, W.W. Hauswirth, T.S. Aleman, S. Kaushal, S.B. Schwartz, S.L. Boye, E.A. Windsor, T.J. Conlon, A. Sumaroka, J.J. Pang, A.J. Roman, B.J. Byrne, and S.G. Jacobson Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year Hum. Gene Ther. 20 2009 999 1004
24. A.V. Cideciyan, S.G. Jacobson, W.A. Beltran, A. Sumaroka, M. Swider, S. Iwabe, A.J. Roman, M.B. Olivares, S.B. Schwartz, A.M. Komaromy, W.W. Hauswirth, and G.D. Aguirre Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement Proc. Natl. Acad. Sci. USA 110 2013 E517 E525
25. F. Testa, A.M. Maguire, S. Rossi, E.A. Pierce, P. Melillo, K. Marshall, S. Banfi, E.M. Surace, J. Sun, C. Acerra, J.F. Wright, J. Wellman, K.A. High, A. Auricchio, J. Bennett, and F. Simonelli Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2 Ophthalmology 120 2013 1283 1291
26. K. Stieger, T. Cronin, J. Bennett, and F. Rolling Adeno-associated virus mediated gene therapy for retinal degenerative diseases Methods Mol. Biol. 807 2011 179 218
27. Z. Wu, H. Yang, and P. Colosi Effect of genome size on AAV vector packaging Mol. Ther. 18 2010 80 86
28. I. Trapani, P. Colella, A. Sommella, C. Iodice, G. Cesi, S. de Simone, E. Marrocco, S. Rossi, M. Giunti, A. Palfi, G.J. Farrar, R. Polishchuk, and A. Auricchio Effective delivery of large genes to the retina by dual AAV vectors EMBO Mol. Med. 6 2014 194 211
29. T.L. Fink, P.J. Klepcyk, S.M. Oette, C.R. Gedeon, S.L. Hyatt, T.H. Kowalczyk, R.C. Moen, and M.J. Cooper Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles Gene Ther. 13 2006 1048 1051
30. M. Weber, J. Rabinowitz, N. Provost, H. Conrath, S. Folliot, D. Briot, Y. Cherel, P. Chenuaud, J. Samulski, P. Moullier, and F. Rolling Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery Mol. Ther. 7 2003 774 781
31. L. Dudus, V. Anand, G.M. Acland, S.J. Chen, J.M. Wilson, K.J. Fisher, A.M. Maguire, and J. Bennett Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV Vis. Res. 39 1999 2545 2553
32. L.E. Rosas, J.L. Grieves, K. Zaraspe, K.M. La Perle, H. Fu, and D.M. McCarty Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity Mol. Ther.: J. Am. Soc. Gene Ther. 20 2012 2098 2110
33. S.M. Conley, and M.I. Naash Nanoparticles for retinal gene therapy Prog. Retin. Eye Res. 29 2010 376 397
34. G.J. Doherty, and H.T. McMahon Mechanisms of endocytosis Annu. Rev. Biochem. 78 2009 857 902
35. A. Verma, and F. Stellacci Effect of surface properties on nanoparticle-cell interactions Small 6 2010 12 21
36. H. Hillaireau, and P. Couvreur Nanocarriers' entry into the cell: relevance to drug delivery Cell. Mol. Life Sci.: CMLS 66 2009 2873 2896
37. C.A. Heth, and M.H. Bernstein Mannose-sensitive HRP endocytosis by the retinal pigment epithelium Exp. Eye Res. 52 1991 75 82
38. S.C. Finnemann, V.L. Bonilha, A.D. Marmorstein, and E. Rodriguez-Boulan Phagocytosis of rod outer segments by retinal pigment epithelial cells requires alpha(v)beta5 integrin for binding but not for internalization Proc. Natl. Acad. Sci. USA 94 1997 12932 12937
39. J.G. Hollyfield, H.H. Varner, M.E. Rayborn, G.I. Liou, and C.D. Bridges Endocytosis and degradation of interstitial retinol-binding protein: differential capabilities of cells that border the interphotoreceptor matrix J. Cell Biol. 100 1985 1676 1681
40. J.G. Hollyfield, and M.E. Rayborn Endocytosis in the inner segment of rod photoreceptors: analysis of Xenopus laevis retinas using horseradish peroxidase Exp. Eye Res. 45 1987 703 719
41. B.R. Bozard, P.S. Ganapathy, J. Duplantier, B. Mysona, Y. Ha, P. Roon, R. Smith, I.D. Goldman, P. Prasad, P.M. Martin, V. Ganapathy, and S.B. Smith Molecular and biochemical characterization of folate transport proteins in retinal Muller cells Invest. Ophthalmol. Vis. Sci. 51 2010 3226 3235
42. B.M. Kevany, and K. Palczewski Phagocytosis of retinal rod and cone photoreceptors Physiology 25 2010 8 15
43. E.F. Nandrot, M. Anand, D. Almeida, K. Atabai, D. Sheppard, and S.C. Finnemann Essential role for MFG-E8 as ligand for alphavbeta5 integrin in diurnal retinal phagocytosis Proc. Natl. Acad. Sci. USA 104 2007 12005 12010
44. P.L. Mayerson, and M.O. Hall Rat retinal pigment epithelial cells show specificity of phagocytosis in vitro J. Cell Biol. 103 1986 299 308
45. D.D. Dunlap, A. Maggi, M.R. Soria, and L. Monaco Nanoscopic structure of DNA condensed for gene delivery Nucleic Acids Res. 25 1997 3095 3101
46. A. Koirala, R.S. Makkia, S.M. Conley, M.J. Cooper, and M.I. Naash S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA Hum. Mol. Genet. 22 2013 1632 1642
47. H.T. McMahon, and E. Boucrot Molecular mechanism and physiological functions of clathrin-mediated endocytosis Nat. Rev. Mol. Cell Biol. 12 2011 517 533
48. S. Xu, B.Z. Olenyuk, C.T. Okamoto, and S.F. Hamm-Alvarez Targeting receptor-mediated endocytotic pathways with nanoparticles: rationale and advances Adv. Drug Deliv. Rev. 65 2013 121 138
49. M.M. Cardoso, I.N. Peca, and A.C. Roque Antibody-conjugated nanoparticles for therapeutic applications Curr. Med. Chem. 19 2012 3103 3127
50. E.C. Cho, J. Xie, P.A. Wurm, and Y. Xia Understanding the role of surface charges in cellular adsorption versus internalization by selectively removing gold nanoparticles on the cell surface with a I2/KI etchant Nano Lett. 9 2009 1080 1084
51. P. Lajoie, and I.R. Nabi Lipid rafts, caveolae, and their endocytosis Int. Rev. Cell Mol. Biol. 282 2010 135 163
52. R.G. Parton, and K. Simons The multiple faces of caveolae Nat. Rev. Mol. Cell Biol. 8 2007 185 194
53. X. Li, M.E. McClellan, M. Tanito, P. Garteiser, R. Towner, D. Bissig, B.A. Berkowitz, S.J. Fliesler, M.L. Woodruff, G.L. Fain, D.G. Birch, M.S. Khan, J.D. Ash, and M.H. Elliott Loss of caveolin-1 impairs retinal function due to disturbance of subretinal microenvironment J. Biol. Chem. 287 2012 16424 16434
54. W.C. Tseng, F.R. Haselton, and T.D. Giorgio Mitosis enhances transgene expression of plasmid delivered by cationic liposomes Biochim. Biophys. Acta 1445 1999 53 64
55. J. Zabner, A.J. Fasbender, T. Moninger, K.A. Poellinger, and M.J. Welsh Cellular and molecular barriers to gene transfer by a cationic lipid J. Biol. Chem. 270 1995 18997 19007
56. M.E. Dowty, P. Williams, G. Zhang, J.E. Hagstrom, and J.A. Wolff Plasmid DNA entry into postmitotic nuclei of primary rat myotubes Proc. Natl. Acad. Sci. USA 92 1995 4572 4576
57. J.A. Wolff, R.W. Malone, P. Williams, W. Chong, G. Acsadi, A. Jani, and P.L. Felgner Direct gene transfer into mouse muscle in vivo Science 247 1990 1465 1468
58. A. Dhanoya, T. Wang, E. Keshavarz-Moore, A. Fassati, and B.M. Chain Importin-7 mediates nuclear trafficking of DNA in mammalian cells Traffic 14 2013 165 175
59. A. Lachish-Zalait, C.K. Lau, B. Fichtman, E. Zimmerman, A. Harel, M.R. Gaylord, D.J. Forbes, and M. Elbaum Transportin mediates nuclear entry of DNA in vertebrate systems Traffic 10 2009 1414 1428
60. A.P. Lam, and D.A. Dean Progress and prospects: nuclear import of nonviral vectors Gene Ther. 17 2010 439 447
61. D.A. Dean Import of plasmid DNA into the nucleus is sequence specific Exp. Cell Res. 230 1997 293 302
62. D.A. Dean, B.S. Dean, S. Muller, and L.C. Smith Sequence requirements for plasmid nuclear import Exp. Cell Res. 253 1999 713 722
63. P. Ghosh, G. Han, M. De, C.K. Kim, and V.M. Rotello Gold nanoparticles in delivery applications Adv. Drug Deliv. Rev. 60 2008 1307 1315
64. R. Levy, U. Shaheen, Y. Cesbron, and V. See Gold nanoparticles delivery in mammalian live cells: a critical review Nano Rev. 1 2010
65. K.K. Ewert, A. Zidovska, A. Ahmad, N.F. Bouxsein, H.M. Evans, C.S. McAllister, C.E. Samuel, and C.R. Safinya Cationic liposome-nucleic acid complexes for gene delivery and silencing: pathways and mechanisms for plasmid DNA and siRNA Top. Curr. Chem. 296 2010 191 226
66. A. Seyfoddin, J. Shaw, and R. Al-Kassas Solid lipid nanoparticles for ocular drug delivery Drug Delivery 17 2010 467 489
67. A. del Pozo-Rodriguez, D. Delgado, A.R. Gascon, and M.A. Solinis Lipid nanoparticles as drug/gene delivery systems to the retina J. Ocul. Pharmacol. Ther.: Off. J. Assoc. Ocul. Pharmacol. Ther. 29 2013 173 188
68. Y. Mo, M.E. Barnett, D. Takemoto, H. Davidson, and U.B. Kompella Human serum albumin nanoparticles for efficient delivery of Cu, Zn superoxide dismutase gene Mol. Vis. 13 2007 746 757
69. A.O. Elzoghby, W.M. Samy, and N.A. Elgindy Albumin-based nanoparticles as potential controlled release drug delivery systems J. Control. Release: Off. J. Control. Release Soc. 157 2012 168 182
70. A.O. Elzoghby Gelatin-based nanoparticles as drug and gene delivery systems: reviewing three decades of research J. Control. Release: Off. J. Control. Release Soc. 172 2013 1075 1091
71. R.N. Mitra, Z. Han, M. Merwin, M. Al Taai, S.M. Conley, and M.I. Naash Synthesis and characterization of glycol chitosan DNA nanoparticles for retinal gene delivery ChemMedChem 9 2014 189 196
72. J. Jin, K.K. Zhou, K. Park, Y. Hu, X. Xu, Z. Zheng, P. Tyagi, U.B. Kompella, and J.X. Ma Anti-inflammatory and antiangiogenic effects of nanoparticle-mediated delivery of a natural angiogenic inhibitor Invest. Ophthalmol. Vis. Sci. 52 2011 6230 6237
73. K. Park, Y. Chen, Y. Hu, A.S. Mayo, U.B. Kompella, R. Longeras, and J.X. Ma Nanoparticle-mediated expression of an angiogenic inhibitor ameliorates ischemia-induced retinal neovascularization and diabetes-induced retinal vascular leakage Diabetes 58 2009 1902 1913
74. K. Raemdonck, T.F. Martens, K. Braeckmans, J. Demeester, and S.C. De Smedt Polysaccharide-based nucleic acid nanoformulations Adv. Drug Deliv. Rev. 65 2013 1123 1147
75. C. Zhang, Y.S. Wang, H. Wu, Z.X. Zhang, Y. Cai, H.Y. Hou, W. Zhao, X.M. Yang, and J.X. Ma Inhibitory efficacy of hypoxia-inducible factor 1alpha short hairpin RNA plasmid DNA-loaded poly (d, l-lactide-co-glycolide) nanoparticles on choroidal neovascularization in a laser-induced rat model Gene Ther. 17 2010 338 351
76. S.R. Singh, H.E. Grossniklaus, S.J. Kang, H.F. Edelhauser, B.K. Ambati, and U.B. Kompella Intravenous transferrin, RGD peptide and dual-targeted nanoparticles enhance anti-VEGF intraceptor gene delivery to laser-induced CNV Gene Ther. 16 2009 645 659
77. F. Danhier, E. Ansorena, J.M. Silva, R. Coco, A. Le Breton, and V. Preat PLGA-based nanoparticles: an overview of biomedical applications J. Control. Release: Off. J. Control. Release Soc. 161 2012 505 522
78. H.K. Makadia, and S.J. Siegel Poly lactic-co-glycolic acid (PLGA) as biodegradable controlled drug delivery carrier Polymers 3 2011 1377 1397
79. R.J. Marano, I. Toth, N. Wimmer, M. Brankov, and P.E. Rakoczy Dendrimer delivery of an anti-VEGF oligonucleotide into the eye: a long-term study into inhibition of laser-induced CNV, distribution, uptake and toxicity Gene Ther. 12 2005 1544 1550
80. H.W. Liao, and K.W. Yau In vivo gene delivery in the retina using polyethylenimine Biotechniques 42 2007 285 286 288
81. S.P. Chaplot, and I.D. Rupenthal Dendrimers for gene delivery - a potential approach for ocular therapy? J. Pharm. Pharmacol. 66 2014 542 556
82. C.C. Lee, J.A. MacKay, J.M. Frechet, and F.C. Szoka Designing dendrimers for biological applications Nat. Biotechnol. 23 2005 1517 1526
83. A. Koirala, S.M. Conley, R. Makkia, Z. Liu, M.J. Cooper, J.R. Sparrow, and M.I. Naash Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases J. Control. Release: Off. J. Control. Release Soc. 172 2013 745 752
84. Z. Han, S.M. Conley, R.S. Makkia, M.J. Cooper, and M.I. Naash DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice J. Clin. Invest. 122 2012 3221 3226
85. X. Cai, Z. Nash, S.M. Conley, S.J. Fliesler, M.J. Cooper, and M.I. Naash A partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticles PLoS One 4 2009 e5290
86. X. Cai, S.M. Conley, Z. Nash, S.J. Fliesler, M.J. Cooper, and M.I. Naash Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa FASEB J. 24 2010 1178 1191
87. S.J. Bakri, J.S. Pulido, P. Mukherjee, R.J. Marler, and D. Mukhopadhyay Absence of histologic retinal toxicity of intravitreal nanogold in a rabbit model Retina 28 2008 147 149
88. A. Hayashi, A. Naseri, M.E. Pennesi, and E. de Juan Jr. Subretinal delivery of immunoglobulin G with gold nanoparticles in the rabbit eye Jpn. J. Ophthalmol. 53 2009 249 256
89. J.H. Kim, J.H. Kim, K.W. Kim, M.H. Kim, and Y.S. Yu Intravenously administered gold nanoparticles pass through the blood-retinal barrier depending on the particle size, and induce no retinal toxicity Nanotechnology 20 2009 505101
90. A.C. Curry, M. Crow, and A. Wax Molecular imaging of epidermal growth factor receptor in live cells with refractive index sensitivity using dark-field microspectroscopy and immunotargeted nanoparticles J. Biomed. Opt. 13 2008 014022
91. G. Louit, T. Asahi, G. Tanaka, T. Uwada, and H. Masuhara Spectral and 3-dimensional tracking of single gold nanoparticles in living cells studied by Rayleigh light scattering microscopy J. Phys. Chem. 113 2009 11766 11772
92. D. Boyer, P. Tamarat, A. Maali, B. Lounis, and M. Orrit Photothermal imaging of nanometer-sized metal particles among scatterers Science 297 2002 1160 1163
93. C. Sonnichsen, B.M. Reinhard, J. Liphardt, and A.P. Alivisatos A molecular ruler based on plasmon coupling of single gold and silver nanoparticles Nat. Biotechnol. 23 2005 741 745
94. K. Fujita, S. Ishitobi, K. Hamada, N.I. Smith, A. Taguchi, Y. Inouye, and S. Kawata Time-resolved observation of surface-enhanced Raman scattering from gold nanoparticles during transport through a living cell J. Biomed. Opt. 14 2009 024038
95. T.M. Mayhew, C. Muhlfeld, D. Vanhecke, and M. Ochs A review of recent methods for efficiently quantifying immunogold and other nanoparticles using TEM sections through cells, tissues and organs Ann. Anat. = Anat. Anz.: Off. Organ Anat. Gesell. 191 2009 153 170
96. M.C. Skala, M.J. Crow, A. Wax, and J.A. Izatt Three-dimensional molecular imaging with photothermal optical coherence tomography Methods Mol. Biol. 1026 2013 85 92
97. L.H. Peng, J. Niu, C.Z. Zhang, W. Yu, J.H. Wu, Y.H. Shan, X.R. Wang, Y.Q. Shen, Z.W. Mao, W.Q. Liang, and J.Q. Gao TAT conjugated cationic noble metal nanoparticles for gene delivery to epidermal stem cells Biomaterials 35 2014 5605 5618
98. P.S. Ghosh, C.K. Kim, G. Han, N.S. Forbes, and V.M. Rotello Efficient gene delivery vectors by tuning the surface charge density of amino acid-functionalized gold nanoparticles ACS Nano 2 2008 2213 2218
99. P.H. Yang, X. Sun, J.F. Chiu, H. Sun, and Q.Y. He Transferrin-mediated gold nanoparticle cellular uptake Bioconjug. Chem. 16 2005 494 496
100. S. Kumar, J. Aaron, and K. Sokolov Directional conjugation of antibodies to nanoparticles for synthesis of multiplexed optical contrast agents with both delivery and targeting moieties Nat. Protoc. 3 2008 314 320
101. W. Eck, G. Craig, A. Sigdel, G. Ritter, L.J. Old, L. Tang, M.F. Brennan, P.J. Allen, and M.D. Mason PEGylated gold nanoparticles conjugated to monoclonal F19 antibodies as targeted labeling agents for human pancreatic carcinoma tissue ACS Nano 2 2008 2263 2272
102. N.D. Sonawane, F.C. Szoka Jr., and A.S. Verkman Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes J. Biol. Chem. 278 2003 44826 44831
103. M. Thomas, and A.M. Klibanov Conjugation to gold nanoparticles enhances polyethylenimine's transfer of plasmid DNA into mammalian cells Proc. Natl. Acad. Sci. USA 100 2003 9138 9143
104. A. Sharma, A. Tandon, J.C. Tovey, R. Gupta, J.D. Robertson, J.A. Fortune, A.M. Klibanov, J.W. Cowden, F.G. Rieger, and R.R. Mohan Polyethylenimine-conjugated gold nanoparticles: gene transfer potential and low toxicity in the cornea Nanomed. Nanotechnol. Biol. Med. 7 2011 505 513
105. P. Hawley-Nelson, G. Ciccarone, Gebeyehu, and J. Jessee Lipofectamine reagent: a new, higher efficiency polycationic liposome transfection reagent Focus 1993 73 79
106. J.O. Radler, I. Koltover, T. Salditt, and C.R. Safinya Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes Science 275 1997 810 814
107. I.S. Zuhorn, R. Kalicharan, and D. Hoekstra Lipoplex-mediated transfection of mammalian cells occurs through the cholesterol-dependent clathrin-mediated pathway of endocytosis J. Biol. Chem. 277 2002 18021 18028
108. J. Rejman, A. Bragonzi, and M. Conese Role of clathrin- and caveolae-mediated endocytosis in gene transfer mediated by lipo- and polyplexes Mol. Ther.: J. Am. Soc. Gene Ther. 12 2005 468 474
109. A. Ahmad, H.M. Evans, K. Ewert, C.X. George, C.E. Samuel, and C.R. Safinya New multivalent cationic lipids reveal bell curve for transfection efficiency versus membrane charge density: lipid-DNA complexes for gene delivery J. Gene Med. 7 2005 739 748
110. K. Ewert, A. Ahmad, H.M. Evans, H.W. Schmidt, and C.R. Safinya Efficient synthesis and cell-transfection properties of a new multivalent cationic lipid for nonviral gene delivery J. Med. Chem. 45 2002 5023 5029
111. K.K. Ewert, H.M. Evans, N.F. Bouxsein, and C.R. Safinya Dendritic cationic lipids with highly charged headgroups for efficient gene delivery Bioconjug. Chem. 17 2006 877 888
112. K.K. Ewert, H.M. Evans, A. Zidovska, N.F. Bouxsein, A. Ahmad, and C.R. Safinya A columnar phase of dendritic lipid-based cationic liposome-DNA complexes for gene delivery: hexagonally ordered cylindrical micelles embedded in a DNA honeycomb lattice J. Am. Chem. Soc. 128 2006 3998 4006
113. I. Masuda, T. Matsuo, T. Yasuda, and N. Matsuo Gene transfer with liposomes to the intraocular tissues by different routes of administration Invest. Ophthalmol. Vis. Sci. 37 1996 1914 1920
114. D. Delgado, A. del Pozo-Rodriguez, M.A. Solinis, M. Aviles-Triqueros, B.H. Weber, E. Fernandez, and A.R. Gascon Dextran and protamine-based solid lipid nanoparticles as potential vectors for the treatment of X-linked juvenile retinoschisis Hum. Gene Ther. 23 2012 345 355
115. S.H. Choi, S.E. Jin, M.K. Lee, S.J. Lim, J.S. Park, B.G. Kim, W.S. Ahn, and C.K. Kim Novel cationic solid lipid nanoparticles enhanced p53 gene transfer to lung cancer cells Eur. J. Pharm. Biopharm.: Off. J. Arbeitsgemeinschaft fur Pharm. Verfahrenstechnik e.V. 68 2008 545 554
116. S. Shi, L. Han, L. Deng, Y. Zhang, H. Shen, T. Gong, Z. Zhang, and X. Sun Dual drugs (microRNA-34a and paclitaxel)-loaded functional solid lipid nanoparticles for synergistic cancer cell suppression J. Control. Release: Off. J. Control. Release Soc. 194C 2014 228 237
117. R. Sandhir, A. Yadav, A. Mehrotra, A. Sunkaria, A. Singh, and S. Sharma Curcumin nanoparticles attenuate neurochemical and neurobehavioral deficits in experimental model of Huntington's disease Neuromol. Med. 16 2014 106 118
118. N. Nafee, A. Husari, C.K. Maurer, C. Lu, C. de Rossi, A. Steinbach, R.W. Hartmann, C.M. Lehr, and M. Schneider Antibiotic-free nanotherapeutics: ultra-small, mucus-penetrating solid lipid nanoparticles enhance the pulmonary delivery and anti-virulence efficacy of novel quorum sensing inhibitors J. Control. Release: Off. J. Control. Release Soc. 192 2014 131 140
119. E. Vighi, E. Leot, M. Montanari, A. Mucci, M. Hanuskova, and V. Iannuccelli Structural investigation and intracellular trafficking of a novel multicomposite cationic solid lipid nanoparticle platform as a pDNA carrier Ther. Deliv. 2 2011 1419 1435
120. S.N. He, Y.L. Li, J.J. Yan, W. Zhang, Y.Z. Du, H.Y. Yu, F.Q. Hu, and H. Yuan Ternary nanoparticles composed of cationic solid lipid nanoparticles, protamine, and DNA for gene delivery Int. J. Nanomed. 8 2013 2859 2869
121. A. del Pozo-Rodriguez, D. Delgado, M.A. Solinis, J.L. Pedraz, E. Echevarria, J.M. Rodriguez, and A.R. Gascon Solid lipid nanoparticles as potential tools for gene therapy: in vivo protein expression after intravenous administration Int. J. Pharm. 385 2010 157 162
122. E. Vighi, B. Ruozi, M. Montanari, R. Battini, and E. Leo PDNA condensation capacity and in vitro gene delivery properties of cationic solid lipid nanoparticles Int. J. Pharm. 389 2010 254 261
123. E. Vighi, M. Montanari, B. Ruozi, G. Tosi, A. Magli, and E. Leo Nuclear localization of cationic solid lipid nanoparticles containing Protamine as transfection promoter Eur. J. Pharm. Biopharm.: Off. J. Arbeitsgemeinschaft fur Pharm. Verfahrenstechnik e.V. 76 2010 384 393
124. A.P. Ruiz de Garibay, M.A. Solinis Aspiazu, A. Rodriguez Gascon, H. Ganjian, and R. Fuchs Role of endocytic uptake in transfection efficiency of solid lipid nanoparticles-based nonviral vectors J. Gene Med. 15 2013 427 440
125. D. Delgado, A.R. Gascon, A. Del Pozo-Rodriguez, E. Echevarria, A.P. Ruiz de Garibay, J.M. Rodriguez, and M.A. Solinis Dextran-protamine-solid lipid nanoparticles as a non-viral vector for gene therapy: in vitro characterization and in vivo transfection after intravenous administration to mice Int. J. Pharm. 425 2012 35 43
126. D. Delgado, A. del Pozo-Rodriguez, M. Angeles Solinis, A. Bartkowiak, and A. Rodriguez-Gascon New gene delivery system based on oligochitosan and solid lipid nanoparticles: 'in vitro' and 'in vivo' evaluation Eur. J. Pharm. Sci.: Off. J. Eur. Fed. Pharm. Sci. 50 2013 484 491
127. C. Rudolph, and J. Rosenecker Formation of solid lipid nanoparticle (SLN)-gene vector complexes for transfection of mammalian cells in vitro Cold Spring Harbor Protoc. 2012 2012 357 360
128. F. Jing, D. Li, W. Xu, Y. Liu, K. Wang, and Z. Sui Transferrin- and folate-modified, double-targeted nanocarriers for gene delivery Pharm. Biol. 52 2014 570 574
129. Y. Han, Y. Zhang, D. Li, Y. Chen, J. Sun, and F. Kong Transferrin-modified nanostructured lipid carriers as multifunctional nanomedicine for codelivery of DNA and doxorubicin Int. J. Nanomed. 9 2014 4107 4116
130. P.S. Apaolaza, D. Delgado, A. del Pozo-Rodriguez, A.R. Gascon, and M.A. Solinis A novel gene therapy vector based on hyaluronic acid and solid lipid nanoparticles for ocular diseases Int. J. Pharm. 465 2014 413 426
131. S. Ganesh, A.K. Iyer, D.V. Morrissey, and M.M. Amiji Hyaluronic acid based self-assembling nanosystems for CD44 target mediated siRNA delivery to solid tumors Biomaterials 34 2013 3489 3502
132. A. del Pozo-Rodriguez, D. Delgado, M.A. Solinis, A.R. Gascon, and J.L. Pedraz Solid lipid nanoparticles for retinal gene therapy: transfection and intracellular trafficking in RPE cells Int. J. Pharm. 360 2008 177 183
133. R. Duncan, and R. Gaspar Nanomedicine(s) under the microscope Mol. Pharm. 8 2011 2101 2141
134. V.L. Truong-Le, J.T. August, and K.W. Leong Controlled gene delivery by DNA-gelatin nanospheres Hum. Gene Ther. 9 1998 1709 1717
135. R.C. Mundargi, V.R. Babu, V. Rangaswamy, P. Patel, and T.M. Aminabhavi Nano/micro technologies for delivering macromolecular therapeutics using poly(d, l-lactide-co-glycolide) and its derivatives J. Control. Release: Off. J. Control. Release Soc. 125 2008 193 209
136. H. Eliyahu, A. Makovitzki, T. Azzam, A. Zlotkin, A. Joseph, D. Gazit, Y. Barenholz, and A.J. Domb Novel dextran-spermine conjugates as transfecting agents: comparing water-soluble and micellar polymers Gene Ther. 12 2005 494 503
137. H. Hosseinkhani, T. Azzam, Y. Tabata, and A.J. Domb Dextran-spermine polycation: an efficient nonviral vector for in vitro and in vivo gene transfection Gene Ther. 11 2004 194 203
138. G.K. Zorzi, J.E. Parraga, B. Seijo, and A. Sanchez Hybrid nanoparticle design based on cationized gelatin and the polyanions dextran sulfate and chondroitin sulfate for ocular gene therapy Macromol. Biosci. 11 2011 905 913
139. F. Danhier, O. Feron, and V. Preat To exploit the tumor microenvironment: passive and active tumor targeting of nanocarriers for anti-cancer drug delivery J. Control. Release: Off. J. Control. Release Soc. 148 2010 135 146
140. S. Rhaese, H. von Briesen, H. Rubsamen-Waigmann, J. Kreuter, and K. Langer Human serum albumin-polyethylenimine nanoparticles for gene delivery J. Control. Release: Off. J. Control. Release Soc. 92 2003 199 208
141. H. Kouchakzadeh, S.A. Shojaosadati, A. Maghsoudi, and E. Vasheghani Farahani Optimization of PEGylation conditions for BSA nanoparticles using response surface methodology AAPS PharmSciTech 11 2010 1206 1211
142. K. Ulbrich, M. Michaelis, F. Rothweiler, T. Knobloch, P. Sithisarn, J. Cinatl, and J. Kreuter Interaction of folate-conjugated human serum albumin (HSA) nanoparticles with tumour cells Int. J. Pharm. 406 2011 128 134
143. K. Ulbrich, T. Hekmatara, E. Herbert, and J. Kreuter Transferrin- and transferrin-receptor-antibody-modified nanoparticles enable drug delivery across the blood-brain barrier (BBB) Eur. J. Pharm. Biopharm.: Off. J. Arbeitsgemeinschaft fur Pharm. Verfahrenstechnik e.V. 71 2009 251 256
144. Z. Li, J. Zhuang, and D.W. Corson Delivery of 9-Cis retinal to photoreceptors from bovine serum albumin Photochem. Photobiol. 69 1999 500 504
145. H.D. Singh, G. Wang, H. Uludag, and L.D. Unsworth Poly-l-lysine-coated albumin nanoparticles: stability, mechanism for increasing in vitro enzymatic resilience, and siRNA release characteristics Acta Biomater. 6 2010 4277 4284
146. S. Zhang, C. Kucharski, M.R. Doschak, W. Sebald, and H. Uludag Polyethylenimine-PEG coated albumin nanoparticles for BMP-2 delivery Biomaterials 31 2010 952 963
147. G. Mayer, V. Vogel, J. Weyermann, D. Lochmann, J.A. van den Broek, C. Tziatzios, W. Haase, D. Wouters, U.S. Schubert, A. Zimmer, J. Kreuter, and D. Schubert Oligonucleotide-protamine-albumin nanoparticles: protamine sulfate causes drastic size reduction J. Control. Release: Off. J. Control. Release Soc. 106 2005 181 187
148. V. Vogel, D. Lochmann, J. Weyermann, G. Mayer, C. Tziatzios, J.A. van den Broek, W. Haase, D. Wouters, U.S. Schubert, J. Kreuter, A. Zimmer, and D. Schubert Oligonucleotide-protamine-albumin nanoparticles: preparation, physical properties, and intracellular distribution J. Control. Release: Off. J. Control. Release Soc. 103 2005 99 111
149. S. Kommareddy, and M. Amiji Preparation and evaluation of thiol-modified gelatin nanoparticles for intracellular DNA delivery in response to glutathione Bioconjug. Chem. 16 2005 1423 1432
150. J. Xu, and M. Amiji Therapeutic gene delivery and transfection in human pancreatic cancer cells using epidermal growth factor receptor-targeted gelatin nanoparticles J. Visualized Exp.: JoVE 2012 e3612
151. J. Xu, A. Singh, and M.M. Amiji Redox-responsive targeted gelatin nanoparticles for delivery of combination wt-p53 expressing plasmid DNA and gemcitabine in the treatment of pancreatic cancer BMC Cancer 14 2014 75
152. S. Kommareddy, and M. Amiji Biodistribution and pharmacokinetic analysis of long-circulating thiolated gelatin nanoparticles following systemic administration in breast cancer-bearing mice J. Pharm. Sci. 96 2007 397 407
153. T. Xia, M. Kovochich, M. Liong, J.I. Zink, and A.E. Nel Cationic polystyrene nanosphere toxicity depends on cell-specific endocytic and mitochondrial injury pathways ACS Nano 2 2008 85 96
154. C.M. Goodman, C.D. McCusker, T. Yilmaz, and V.M. Rotello Toxicity of gold nanoparticles functionalized with cationic and anionic side chains Bioconjug. Chem. 15 2004 897 900
155. G. Konat Zorzi, L. Contreras-Ruiz, J.E. Parraga, A. Lopez-Garcia, R. Romero Bello, Y. Diebold, B. Seijo, and A. Sanchez Expression of MUC5AC in ocular surface epithelial cells using cationized gelatin nanoparticles Mol. Pharm. 8 2011 1783 1788
156. L. Contreras-Ruiz, G.K. Zorzi, D. Hileeto, A. Lopez-Garcia, M. Calonge, B. Seijo, A. Sanchez, and Y. Diebold A nanomedicine to treat ocular surface inflammation: performance on an experimental dry eye murine model Gene Ther. 20 2013 467 477
157. S. Abdullah, W.Y. Wendy-Yeo, H. Hosseinkhani, M. Hosseinkhani, E. Masrawa, R. Ramasamy, R. Rosli, S.A. Rahman, and A.J. Domb Gene transfer into the lung by nanoparticle dextran-spermine/plasmid DNA complexes J. Biomed. Biotechnol. 2010 2010 284840
158. F. Abedini, H. Hosseinkhani, M. Ismail, A.J. Domb, A.R. Omar, P.P. Chong, P.D. Hong, D.S. Yu, and I.Y. Farber Cationized dextran nanoparticle-encapsulated CXCR4-siRNA enhanced correlation between CXCR4 expression and serum alkaline phosphatase in a mouse model of colorectal cancer Int. J. Nanomed. 7 2012 4159 4168
159. Y. Onishi, Y. Eshita, A. Murashita, M. Mizuno, and J. Yoshida Characteristics of DEAE-dextran-MMA graft copolymer as a nonviral gene carrier Nanomed. Nanotechnol. Biol. Med. 3 2007 184 191
160. J.A. Cohen, T.T. Beaudette, J.L. Cohen, K.E. Broaders, E.M. Bachelder, and J.M. Frechet Acetal-modified dextran microparticles with controlled degradation kinetics and surface functionality for gene delivery in phagocytic and non-phagocytic cells Adv. Mater. 22 2010 3593 3597
161. J.C. Kasper, D. Schaffert, M. Ogris, E. Wagner, and W. Friess Development of a lyophilized plasmid/LPEI polyplex formulation with long-term stability - a step closer from promising technology to application J. Control. Release: Off. J. Control. Release Soc. 151 2011 246 255
162. C. Moreira, H. Oliveira, L.R. Pires, S. Simoes, M.A. Barbosa, and A.P. Pego Improving chitosan-mediated gene transfer by the introduction of intracellular buffering moieties into the chitosan backbone Acta Biomater. 5 2009 2995 3006
163. S. Mao, W. Sun, and T. Kissel Chitosan-based formulations for delivery of DNA and siRNA Adv. Drug Deliv. Rev. 62 2010 12 27
164. B. Ghosn, S.P. Kasturi, and K. Roy Enhancing polysaccharide-mediated delivery of nucleic acids through functionalization with secondary and tertiary amines Curr. Top. Med. Chem. 8 2008 331 340
165. B. Ghosn, A. Singh, M. Li, A.V. Vlassov, C. Burnett, N. Puri, and K. Roy Efficient gene silencing in lungs and liver using imidazole-modified chitosan as a nanocarrier for small interfering RNA Oligonucleotides 20 2010 163 172
166. K.A. Athanasiou, G.G. Niederauer, and C.M. Agrawal Sterilization, toxicity, biocompatibility and clinical applications of polylactic acid/polyglycolic acid copolymers Biomaterials 17 1996 93 102
167. B. Semete, L. Booysen, Y. Lemmer, L. Kalombo, L. Katata, J. Verschoor, and H.S. Swai In vivo evaluation of the biodistribution and safety of PLGA nanoparticles as drug delivery systems Nanomed. Nanotechnol. Biol. Med. 6 2010 662 671
168. J. Panyam, W.Z. Zhou, S. Prabha, S.K. Sahoo, and V. Labhasetwar Rapid endo-lysosomal escape of poly(d, l-lactide-co-glycolide) nanoparticles: implications for drug and gene delivery FASEB J. 16 2002 1217 1226
169. K. Tahara, T. Sakai, H. Yamamoto, H. Takeuchi, and Y. Kawashima Establishing chitosan coated PLGA nanosphere platform loaded with wide variety of nucleic acid by complexation with cationic compound for gene delivery Int. J. Pharm. 354 2008 210 216
170. I.S. Kim, S.K. Lee, Y.M. Park, Y.B. Lee, S.C. Shin, K.C. Lee, and I.J. Oh Physicochemical characterization of poly(l-lactic acid) and poly(d, l-lactide-co-glycolide) nanoparticles with polyethylenimine as gene delivery carrier Int. J. Pharm. 298 2005 255 262
171. N. Karra, T. Nassar, A.N. Ripin, O. Schwob, J. Borlak, and S. Benita Antibody conjugated PLGA nanoparticles for targeted delivery of paclitaxel palmitate: efficacy and biofate in a lung cancer mouse model Small 9 2013 4221 4236
172. J.L. Bourges, S.E. Gautier, F. Delie, R.A. Bejjani, J.C. Jeanny, R. Gurny, D. BenEzra, and F.F. Behar-Cohen Ocular drug delivery targeting the retina and retinal pigment epithelium using polylactide nanoparticles Invest. Ophthalmol. Vis. Sci. 44 2003 3562 3569
173. S. Omri, B. Omri, M. Savoldelli, L. Jonet, B. Thillaye-Goldenberg, G. Thuret, P. Gain, J.C. Jeanny, P. Crisanti, and F. Behar-Cohen The outer limiting membrane (OLM) revisited: clinical implications Clin. Ophthalmol. 4 2010 183 195
174. J.D. Eichman, A.U. Bielinska, J.F. Kukowska-Latallo, and J.R. Baker Jr. The use of PAMAM dendrimers in the efficient transfer of genetic material into cells Pharm. Sci. Technol. Today 3 2000 232 245
175. H. Arima, M. Arizono, T. Higashi, A. Yoshimatsu, H. Ikeda, K. Motoyama, K. Hattori, T. Takeuchi, F. Hirayama, and K. Uekama Potential use of folate-polyethylene glycol (PEG)-appended dendrimer (G3) conjugate with alpha-cyclodextrin as DNA carriers to tumor cells Cancer Gene Ther. 19 2012 358 366
176. M. Mannisto, S. Vanderkerken, V. Toncheva, M. Elomaa, M. Ruponen, E. Schacht, and A. Urtti Structure-activity relationships of poly(l-lysines): effects of pegylation and molecular shape on physicochemical and biological properties in gene delivery J. Control. Release: Off. J. Control. Release Soc. 83 2002 169 182
177. J. Intra, and A.K. Salem Characterization of the transgene expression generated by branched and linear polyethylenimine-plasmid DNA nanoparticles in vitro and after intraperitoneal injection in vivo J. Control. Release: Off. J. Control. Release Soc. 130 2008 129 138
178. V. Russ, M. Gunther, A. Halama, M. Ogris, and E. Wagner Oligoethylenimine-grafted polypropylenimine dendrimers as degradable and biocompatible synthetic vectors for gene delivery J. Control. Release: Off. J. Control. Release Soc. 132 2008 131 140
179. R.Q. Huang, Y.H. Qu, W.L. Ke, J.H. Zhu, Y.Y. Pei, and C. Jiang Efficient gene delivery targeted to the brain using a transferrin-conjugated polyethyleneglycol-modified polyamidoamine dendrimer FASEB J.: Off. Publ. Fed. Am. Soc. Exp. Biol. 21 2007 1117 1125
180. O. Boussif, F. Lezoualc'h, M.A. Zanta, M.D. Mergny, D. Scherman, B. Demeneix, and J.P. Behr A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine Proc. Natl. Acad. Sci. USA 92 1995 7297 7301
181. J.F. Kukowska-Latallo, A.U. Bielinska, J. Johnson, R. Spindler, D.A. Tomalia, and J.R. Baker Jr. Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers Proc. Natl. Acad. Sci. USA 93 1996 4897 4902
182. S. Yang, D.J. Coles, A. Esposito, D.J. Mitchell, I. Toth, and R.F. Minchin Cellular uptake of self-assembled cationic peptide-DNA complexes: multifunctional role of the enhancer chloroquine J. Control. Release: Off. J. Control. Release Soc. 135 2009 159 165
183. G. Navarro, and C. Tros de Ilarduya Activated and non-activated PAMAM dendrimers for gene delivery in vitro and in vivo Nanomed. Nanotechnol. Biol. Med. 5 2009 287 297
184. J.S. Choi, K. Nam, J.Y. Park, J.B. Kim, J.K. Lee, and J.S. Park Enhanced transfection efficiency of PAMAM dendrimer by surface modification with l-arginine J. Control. Release: Off. J. Control. Release Soc. 99 2004 445 456
185. J.L. Santos, D. Pandita, J. Rodrigues, A.P. Pego, P.L. Granja, G. Balian, and H. Tomas Receptor-mediated gene delivery using PAMAM dendrimers conjugated with peptides recognized by mesenchymal stem cells Mol. Pharm. 7 2010 763 774
186. Z. Yin, N. Liu, M. Ma, L. Wang, Y. Hao, and X. Zhang A novel EGFR-targeted gene delivery system based on complexes self-assembled by EGF, DNA, and activated PAMAM dendrimers Int. J. Nanomed. 7 2012 4625 4635
187. L. Albertazzi, L. Gherardini, M. Brondi, S. Sulis Sato, A. Bifone, T. Pizzorusso, G.M. Ratto, and G. Bardi In vivo distribution and toxicity of PAMAM dendrimers in the central nervous system depend on their surface chemistry Mol. Pharm. 10 2013 249 260
188. C. Rudolph, J. Lausier, S. Naundorf, R.H. Muller, and J. Rosenecker In vivo gene delivery to the lung using polyethylenimine and fractured polyamidoamine dendrimers J. Gene Med. 2 2000 269 278
189. M.E. Bonnet, P. Erbacher, and A.L. Bolcato-Bellemin Systemic delivery of DNA or siRNA mediated by linear polyethylenimine (L-PEI) does not induce an inflammatory response Pharm. Res. 25 2008 2972 2982
190. Z. Tang, P. Arjunan, C. Lee, Y. Li, A. Kumar, X. Hou, B. Wang, P. Wardega, F. Zhang, L. Dong, Y. Zhang, S.Z. Zhang, H. Ding, R.N. Fariss, K.G. Becker, J. Lennartsson, N. Nagai, Y. Cao, and X. Li Survival effect of PDGF-CC rescues neurons from apoptosis in both brain and retina by regulating GSK3beta phosphorylation J. Exp. Med. 207 2010 867 880
191. X. Chen, D.M. Kube, M.J. Cooper, and P.B. Davis Cell surface nucleolin serves as receptor for DNA nanoparticles composed of pegylated polylysine and DNA Mol. Ther. 16 2008 333 342
192. X. Chen, S. Shank, P.B. Davis, and A.G. Ziady Nucleolin-mediated cellular trafficking of DNA nanoparticle is lipid raft and microtubule dependent and can be modulated by glucocorticoid Mol. Ther. 19 2011 93 102
193. A.G. Ziady, C.R. Gedeon, T. Miller, W. Quan, J.M. Payne, S.L. Hyatt, T.L. Fink, O. Muhammad, S. Oette, T. Kowalczyk, M.K. Pasumarthy, R.C. Moen, M.J. Cooper, and P.B. Davis Transfection of airway epithelium by stable PEGylated poly-l-lysine DNA nanoparticles in vivo Mol. Ther. 8 2003 936 947
194. X.Q. Ding, A.B. Quiambao, J.B. Fitzgerald, M.J. Cooper, S.M. Conley, and M.I. Naash Ocular delivery of compacted DNA-nanoparticles does not elicit toxicity in the mouse retina PLoS One 4 2009 e7410
195. Z. Han, A. Koirala, R. Makkia, M.J. Cooper, and M.I. Naash Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: expression, repeat delivery and lack of toxicity Nanomedicine (Lond.) 7 2012 521 539
196. L. Li, J. Hou, X. Liu, Y. Guo, Y. Wu, L. Zhang, and Z. Yang Nucleolin-targeting liposomes guided by aptamer AS1411 for the delivery of siRNA for the treatment of malignant melanomas Biomaterials 35 2014 3840 3850
197. Q. Hu, G. Gu, Z. Liu, M. Jiang, T. Kang, D. Miao, Y. Tu, Z. Pang, Q. Song, L. Yao, H. Xia, H. Chen, X. Jiang, X. Gao, and J. Chen F3 peptide-functionalized PEG-PLA nanoparticles co-administrated with tLyp-1 peptide for anti-glioma drug delivery Biomaterials 34 2013 1135 1145
198. M.M. Lufino, P.A. Edser, and R. Wade-Martins Advances in high-capacity extrachromosomal vector technology: episomal maintenance, vector delivery, and transgene expression Mol. Ther.: J. Am. Soc. Gene Ther. 16 2008 1525 1538
199. E.P. Papapetrou, P.G. Ziros, I.D. Micheva, N.C. Zoumbos, and A. Athanassiadou Gene transfer into human hematopoietic progenitor cells with an episomal vector carrying an S/MAR element Gene Ther. 13 2006 40 51
200. C. Piechaczek, C. Fetzer, A. Baiker, J. Bode, and H.J. Lipps A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells Nucleic Acids Res. 27 1999 426 428
201. O. Argyros, S.P. Wong, M. Niceta, S.N. Waddington, S.J. Howe, C. Coutelle, A.D. Miller, and R.P. Harbottle Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector Gene Ther. 15 2008 1593 1605
202. J.H. Martens, M. Verlaan, E. Kalkhoven, J.C. Dorsman, and A. Zantema Scaffold/matrix attachment region elements interact with a p300-scaffold attachment factor A complex and are bound by acetylated nucleosomes Mol. Cell. Biol. 22 2002 2598 2606
203. B.H. Jenke, C.P. Fetzer, I.M. Stehle, F. Jonsson, F.O. Fackelmayer, H. Conradt, J. Bode, and H.J. Lipps An episomally replicating vector binds to the nuclear matrix protein SAF-A in vivo EMBO Rep. 3 2002 349 354
204. J. Bode, Y. Kohwi, L. Dickinson, T. Joh, D. Klehr, C. Mielke, and T. Kohwi-Shigematsu Biological significance of unwinding capability of nuclear matrix-associating DNAs Science 255 1992 195 197
205. S. Rupprecht, and H.J. Lipps Cell cycle dependent histone dynamics of an episomal non-viral vector Gene 439 2009 95 101
206. A. Koirala, S.M. Conley, and M.I. Naash Episomal maintenance of S/MAR-containing non-viral vectors for RPE-based diseases Adv. Exp. Med. Biol. 801 2014 703 709
207. D. Schaarschmidt, J. Baltin, I.M. Stehle, H.J. Lipps, and R. Knippers An episomal mammalian replicon: sequence-independent binding of the origin recognition complex EMBO J. 23 2004 191 201
208. A.C. Jenke, T. Eisenberger, A. Baiker, I.M. Stehle, S. Wirth, and H.J. Lipps The nonviral episomal replicating vector pEPI-1 allows long-term inhibition of bcr-abl expression by shRNA Hum. Gene Ther. 16 2005 533 539
209. D.J. Zack, J. Bennett, Y. Wang, C. Davenport, B. Klaunberg, J. Gearhart, and J. Nathans Unusual topography of bovine rhodopsin promoter-lacZ fusion gene expression in transgenic mouse retinas Neuron 6 1991 187 199
210. J.E. Young, T. Vogt, K.W. Gross, and S.C. Khani A short, highly active photoreceptor-specific enhancer/promoter region upstream of the human rhodopsin kinase gene Invest. Ophthalmol. Vis. Sci. 44 2003 4076 4085
211. A. Furukawa, C. Koike, P. Lippincott, C.L. Cepko, and T. Furukawa The mouse Crx 5′-upstream transgene sequence directs cell-specific and developmentally regulated expression in retinal photoreceptor cells J. Neurosci. Off. J. Soc. Neurosci. 22 2002 1640 1647
212. G.I. Liou, L. Geng, M.R. al-Ubaidi, S. Matragoon, G. Hanten, W. Baehr, and P.A. Overbeek Tissue-specific expression in transgenic mice directed by the 5′-flanking sequences of the human gene encoding interphotoreceptor retinoid-binding protein J. Biol. Chem. 265 1990 8373 8376
213. A.M. Miller, and D.A. Dean Tissue-specific and transcription factor-mediated nuclear entry of DNA Adv. Drug Deliv. Rev. 61 2009 603 613
214. Y. Fei, and T.E. Hughes Nuclear trafficking of photoreceptor protein crx: the targeting sequence and pathologic implications Invest. Ophthalmol. Vis. Sci. 41 2000 2849 2856
215. S. Genini, W.A. Beltran, and G.D. Aguirre Up-regulation of tumor necrosis factor superfamily genes in early phases of photoreceptor degeneration PLoS One 8 2013 e85408
216. M. Thiersch, W. Raffelsberger, R. Frigg, M. Samardzija, A. Wenzel, O. Poch, and C. Grimm Analysis of the retinal gene expression profile after hypoxic preconditioning identifies candidate genes for neuroprotection BMC Genomics 9 2008 73
217. J.Y. Qin, L. Zhang, K.L. Clift, I. Hulur, A.P. Xiang, B.Z. Ren, and B.T. Lahn Systematic comparison of constitutive promoters and the doxycycline-inducible promoter PLoS One 5 2010 e10611
218. L. Yin, K. Greenberg, J.J. Hunter, D. Dalkara, K.D. Kolstad, B.D. Masella, R. Wolfe, M. Visel, D. Stone, R.T. Libby, D. Diloreto Jr., D. Schaffer, J. Flannery, D.R. Williams, and W.H. Merigan Intravitreal injection of AAV2 transduces macaque inner retina Invest. Ophthalmol. Vis. Sci. 52 2011 2775 2783
219. M. Allocca, C. Mussolino, M. Garcia-Hoyos, D. Sanges, C. Iodice, M. Petrillo, L.H. Vandenberghe, J.M. Wilson, V. Marigo, E.M. Surace, and A. Auricchio Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors J. Virol. 81 2007 11372 11380
220. B.J. Raisler, K.I. Berns, M.B. Grant, D. Beliaev, and W.W. Hauswirth Adeno-associated virus type-2 expression of pigmented epithelium-derived factor or Kringles 1-3 of angiostatin reduce retinal neovascularization Proc. Natl. Acad. Sci. USA 99 2002 8909 8914
221. A.R. Brooks, R.N. Harkins, P. Wang, H.S. Qian, P. Liu, and G.M. Rubanyi Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle J. Gene Med. 6 2004 395 404
222. Z.Y. Chen, E. Riu, C.Y. He, H. Xu, and M.A. Kay Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation Mol. Ther. 16 2008 548 556
223. M.J. Pikaart, F. Recillas-Targa, and G. Felsenfeld Loss of transcriptional activity of a transgene is accompanied by DNA methylation and histone deacetylation and is prevented by insulators Genes Dev. 12 1998 2852 2862
224. J.E. Phillips, and V.G. Corces CTCF: master weaver of the genome Cell 137 2009 1194 1211
225. L.E. Gracey Maniar, J.M. Maniar, Z.Y. Chen, J. Lu, A.Z. Fire, and M.A. Kay Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level Mol. Ther.: J. Am. Soc. Gene Ther. 21 2013 131 138
226. Z.Y. Chen, C.Y. He, A. Ehrhardt, and M.A. Kay Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo Mol. Ther. 8 2003 495 500
227. W.M. Dietz, N.E. Skinner, S.E. Hamilton, M.D. Jund, S.M. Heitfeld, A.J. Litterman, P. Hwu, Z.Y. Chen, A.M. Salazar, J.R. Ohlfest, B.R. Blazar, C.A. Pennell, and M.J. Osborn Minicircle DNA is superior to plasmid DNA in eliciting antigen-specific CD8+ T-cell responses Mol. Ther.: J. Am. Soc. Gene Ther. 21 2013 1526 1535
228. S. Stenler, A. Andersson, O.E. Simonson, K.E. Lundin, Z.Y. Chen, M.A. Kay, C.I. Smith, C. Sylven, and P. Blomberg Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor J. Cardiovasc. Pharmacol. 53 2009 18 23
229. O. Argyros, S.P. Wong, C. Fedonidis, O. Tolmachov, S.N. Waddington, S.J. Howe, M. Niceta, C. Coutelle, and R.P. Harbottle Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver J. Mol. Med. (Berl.) 89 2011 515 529
230. C. Hagedorn, M.N. Antoniou, and H.J. Lipps Genomic cis-acting sequences improve expression and establishment of a nonviral vector, molecular therapy Nucleic Acids 2 2013 e118
231. R. Haase, O. Argyros, S.P. Wong, R.P. Harbottle, H.J. Lipps, M. Ogris, T. Magnusson, M.G. Vizoso Pinto, J. Haas, and A. Baiker pEPito: a significantly improved non-viral episomal expression vector for mammalian cells BMC Biotechnol. 10 2010 20
232. J.E. Loeb, W.S. Cordier, M.E. Harris, M.D. Weitzman, and T.J. Hope Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy Hum. Gene Ther. 10 1999 2295 2305
233. K.R. Martin, R.L. Klein, and H.A. Quigley Gene delivery to the eye using adeno-associated viral vectors Methods 28 2002 267 275
234. K.R. Martin, H.A. Quigley, D.J. Zack, H. Levkovitch-Verbin, J. Kielczewski, D. Valenta, L. Baumrind, M.E. Pease, R.L. Klein, and W.W. Hauswirth Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model Invest. Ophthalmol. Vis. Sci. 44 2003 4357 4365
235. D.S. Cross, and J.K. Burmester Functional characterization of the GDEP promoter and three enhancer elements in retinoblastoma and prostate cell lines Med. Oncol. 25 2008 40 49
236. S. Fukunaga, G. Kanda, J. Tanase, H. Harashima, T. Ohyama, and H. Kamiya A designed curved DNA sequence remarkably enhances transgene expression from plasmid DNA in mouse liver Gene Ther. 19 2012 828 835
237. N. Sumida, J. Nishikawa, H. Kishi, M. Amano, T. Furuya, H. Sonobe, and T. Ohyama A designed curved DNA segment that is a remarkable activator of eukaryotic transcription FEBS J. 273 2006 5691 5702