Corneal Gene therapy: Basic science and translational perspective

Ocular Surface

Volumn 11, Issue 3, 2013, Pages 150-164

  Mohan, Rajiv R ^a,b   Rodier, Jason T ^a,b   Sharma, Ajay ^a,b  

^a VETERANS AFFAIRS MEDICAL CENTER   (United States)

^b University of Missouri   (United States)

Author keywords

Adeno associated virus; Adenovirus; Cornea; Corneal diseases and dystrophies; Gene therapy; Lentivirus; Nanoparticles; Retrovirus

Indexed keywords

ADENOVIRUS VECTOR; LENTIVIRUS VECTOR; NANOPARTICLE; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; VIRUS VECTOR;

ARTICLE; BLINDNESS; CAUSTIC BURN; CORNEA; CORNEA DYSTROPHY; CORNEA NEOVASCULARIZATION; CORNEA TRANSPLANTATION; GENE TARGETING; GENE THERAPY; GENE VECTOR; HUMAN; NONHUMAN; OCULAR SURFACE DISEASE; WOUND HEALING;

EID: 84884679402     PISSN: 15420124     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jtos.2012.10.004     Document Type: Review

References (96)

1. Volpers C, Kochanek S. Adenoviral vectors for gene transfer and therapy. J Gene Med 2004;6:S164-71
2. Fehervari Z, Rayner SA, Oral HB, et al. Gene transfer to ex vivo stored corneas. Cornea 1997;16:459-64
3. Mohan RR, Tovey J, Sharma A, Tandon A. Gene therapy in the cornea: 2005-present. Prog Retin Eye Res 2012;31:43-64
4. Mohan RR, Sharma A, Netto MV, et al. Gene therapy in the cornea. Prog Retin Eye Res 2005;24:537-59
5. Mashhour B, Couton D, Perricaudet M, Briand P. In vivo adenovirusmediated gene transfer into ocular tissues. Gene Ther 1994;1:122-6
6. Larkin DF, Oral HB, Ring CJ, et al. Adenovirus-mediated gene delivery to the corneal endothelium. Transplantation 1996;61:363-70
7. Klebe S, Sykes PJ, Coster DJ, et al. Prolongation of sheep corneal allograft survival by ex vivo transfer of the gene encoding interleukin-10. Transplantation 2001;71:1214-20
8. Borras T, Tamm ER, Zigler Jr JS. Ocular adenovirus gene transfer varies in efficiency and inflammatory response. Invest Ophthalmol Vis Sci 1996;37:1282-93
9. Borras T, Gabelt BT, Klintworth GK, et al. Non-invasive observation of repeated adenoviral GFP gene delivery to the anterior segment of the monkey eye in vivo. J Gene Med 2001;3:437-49
10. Budenz DL, Bennett J, Alonso L. In vivo gene transfer into murine corneal endothelial and trabecular meshwork cells. Invest Ophthalmol Vis Sci 1995;36:2211-5
11. Carlson EC, Liu CY, Yang X. In vivo gene delivery and visualization of corneal stromal cells using an adenoviral vector and keratocyte-specific promoter. Invest Ophthalmol Vis Sci 2004;45:2194-200
12. Tsubota K, Inoue H, Ando K, et al. Adenovirus-mediated gene transfer to the ocular surface epithelium. Exp Eye Res 1998;67:531-8
13. Mohan RR, Possin DE, Mohan RR, et al. Development of genetically engineered tet HPV16-E6/E7 transduced human corneal epithelial clones having tight regulation of proliferation and normal differentiation. Exp Eye Res 2003;76:373-407
14. Jester JV, Huang J, Fischer S, et al. Myofibroblast differentiation of normal human keratocytes and hTERT, extended-life human corneal fibroblasts. Invest Ophthalmol Vis Sci 2003;44:1850-8
15. Wilson SE, Weng J, Blair S, et al. Expression of E6/E7 or SV40 large T antigen-coding oncogenes in human corneal endothelial cells indicates regulated high-proliferative capacity. Invest Ophthalmol Vis Sci 1995;36:32-40
16. He Y, Weng J, Li Q, et al. Fuchs' corneal endothelial cells transduced with the human papilloma virus E6/E7 oncogenes. Exp Eye Res 1997;65:135-42
17. Seitz B, Moreira L, Baktanian E, et al. Retroviral vector-mediated gene transfer into keratocytes in vitro and in vivo. Am J Ophthalmo 1998;126:630-9
18. Seitz B, Baktanian E, Gordon EM, et al. Retroviral vector-mediated gene transfer into keratocytes: in vitro effects of polybrene and protamine sulfate. Graefes Arch Clin Exp Ophthalmol 1998;236:602-12
19. Miyoshi H, Blomer U, Takahasi M, et al. Development of a self-inactivating lentivirus vector. J Virol 1998;72:8150-7
20. Wang X, Appukuttan B, Ott S, et al. Efficient and sustained transgene expression in human corneal cells mediated by a lentiviral vectors. Gene Ther 2000;7:196-200
21. Bainbridge JW, Stephens C, Parsley K, et al. In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium. Gene Ther 2001;8:1665-8
22. Challa P, Luna C, Liton PB, et al. Lentiviral mediated gene delivery to the anterior chamber of rodent eyes. Mol Vis 2005;11:425-30
23. Takahashi K, Luo T, Saishin Y, et al. Sustained transduction of ocular cells with a bovine immunodeficiency viral vector. Hum Gene Ther 2002;13:1305-16
24. Beutelspacher SC, Ardjomand N, Tan PH, et al. Comparison of HIV-1 and EIAV-based lentiviral vectors in corneal transduction. Exp Eye Res 2005;80:787-94
25. Parker DG, Kaufmann C, Brereton HM, et al. Lentivirus-mediated gene transfer to the rat, ovine and human cornea. Gene Ther 2007;14:760-7
26. Mohan RR, Schultz GS, Sinha S, et al. Controlled and site-selective gene delivery into mouse keratocytes by adeno-associated viral, lentiviral and plasmid vectors. Inves Ophthalmol Vis Sci 2005;46:2163. ARVO abstract
27. Grimm D, Kay MA, Kleinschmidt JA. Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther 2003;7:839-50
28. Alexander JJ, Hauswirth WW. Adeno-associated viral vectors and the retina. Adv Exp Med Biol 2008;613:121-8
29. Gao G, Vandenberghe LH, Wilson JM. New recombinant serotypes of AAV vectors. Curr Gene Ther 2005;5:285-97
30. Vandenberghe LH, Auricchio A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther 2012;19:162-8
31. Mohan RR, Schults GS, Hong JW, et al. Gene transfer into rabbit keratocytes using AAV and lipid-mediated plasmid DNA vectors with a lamellar flap for stromal access. Exp Eye Res 2003;76:373-83
32. Stieger K, Cronin T, Bennett J, Rolling F. Adeno-associated virus mediated gene therapy for retinal degenerative diseases. Methods Mol Biol 2011;807:179-218
33. Buss DG, Giuliano E, Sharma A, Mohan RR. Gene delivery in the equine cornea: a novel therapeutic strategy. Vet Ophthalmol 2010;13:301-6
34. Lebherz C, Maguire A, Tang W, et al. Novel AAV serotypes for improved ocular gene transfer. J Gene Med 2008;10:375-82
35. Mohan RR, Sinha S, Tandon A, et al. Efficacious and safe tissueselective controlled gene therapy approaches for the cornea. PLoS One 2011;6:e18771
36. Sharma A, Ghosh A, Siddappa C, Mohan RR. Ocular surface: gene therapy. In: Besharse J, Dana R, Dartt DA, eds. Encyclopedia of the eye. Elsevier, 2010. p. 185-94
37. Sharma A, Ghosh A, Hansen ET, et al. Transduction efficiency of AAV 2/6, 2/8 and 2/9 vectors for delivering genes in human corneal fibroblasts. Brain Res Bull 2010;81:273-8
38. Sharma A, Tovey JC, Ghosh A, Mohan RR. AAV serotype influences gene transfer in corneal stroma in vivo. Exp Eye Res 2010;91:440-8
39. Buie LK, Rasmussen CA, Porterfield EC, et al. Self-complementary AAV virus (scAAV) safe and long-term gene transfer in the trabecular meshwork of living rats and monkeys. Invest Ophthalmol Vis Sci 2010;51:236-48
40. Kong F, Li W, Li X, Zheng Q, et al. Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse. Exp Eye Res 2010;90:546-54
41. Yokoi K, Kachi S, Zhang HS, et al. Ocular gene transfer with self-complementary AAV vectors. Invest Ophthalmol Vis Sci 2007;48:3324-8
42. Van Vliet KM, Blouin V, Brument N, et al. The role of the adeno-associated virus capsid in gene transfer. Methods Mol Biol 2008;437:51-91
43. Petrs-Silva H, Dinculescu A, Li Q, et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 2009;17:463-71
44. Zhong L, Li B, Jayandharan G, et al. Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression. Virology 2008;381:194-202
45. Ryals RC, Boye SL, Dinculescu A, et al. Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines. Mol Vis 2011;17:1090-102
46. Sharma A, Tovey J C K, Hauswirth WW, et al. Remarkably high and selective transgene delivery into mouse corneal endothelium in vivo with tyrosine-mutant AAV8. Invest Ophthalmol Vis Sci 2010; ARVO 51:2839
47. Hart SL, Arancibia-Carcamo CV, Wolfert MA, et al. Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector. Hum Gene Ther 1998;9:575-85
48. Angella GJ, Sherwood MB, Balasubramanian L, et al. Enhanced shortterm plasmid transfection of filtration surgery tissues. Invest Ophthalmol Vis Sci 2000;41:4158-62
49. Yoon KC, Ahn KY, Lee JH, et al. Lipid-mediated delivery of brainspecific angiogenesis inhibitor 1 gene reduces corneal neovascularization in an in vivo rabbit model. Gene Ther 2005;12:617-24
50. Pleyer U, Dannowski H. Delivery of genes via liposomes to corneal endothelial cells. Drug News Perspect 2002;15:283-9
51. Toropainen E, Hornof M, Kaarniranta K, et al. Corneal epithelium as a platform for secretion of transgene products after transfection with liposomal gene eyedrops. J Gene Med 2007;9:208-16
52. Dannowski H, Bednarz J, Reszka R, et al. Lipid-mediated gene transfer of acidic fibroblast growth factor into human corneal endothelial cells. Exp Eye Res 2005;80:93-101
53. Oshima Y, Sakamoto T, Tamanaka I, et al. Targeted gene transfer to corneal endothelium in vivo by electric pulse. Gene Ther 1998;5:1347-54
54. Oshima Y, Sakamoto T, Hisatomi T, et al. Targeted gene transfer to corneal stroma in vivo by electric pulses. Exp. Eye Res 2002;74:191-8
55. Zhou R, Dean DA. Gene transfer of interleukin 10 to the murine cornea using electroporation. Exp Biol Med (Maywood) 2007;232:362-9
56. He Z, Pipparelli A, Manissolle C, et al. Ex vivo gene electrotransfer to the endothelium of organ cultured human corneas. Ophthalmic Res 2010;43:43-55
57. Korampally M, Apperson SJ, Staley CS, et al. Transient pressure mediated intranuclear delivery of FITC-Dextran into chicken cardiomyocytes by MEMS-based nanothermite reaction actuator. Sensors and Actuators B: Chemical 2012;171-172:1292-6 http://dx.doi.org/10.1016/j.snb.2012.06.081
58. Mitragotri S. Healing sound: the use of ultrasound in drug delivery and other therapeutic applications. Nat Rev Drug Discov 2005;4:255-60
59. Sonoda S, Tachibana K, Uchino E, et al. Gene transfer to corneal epithelium and keratocytes mediated by ultrasound with microbubbles. Invest Ophthalmol Vis Sci 2006;47:558-64
60. Gupta R, Tandon A, Hansen ET, et al. Rapid and substantial gene delivery into cornea in vivo and in vitro with linearized polyethyleneimine nanoparticles. Invest Ophthalmol Vis Sci 2011;52:494. ARVO E-abstract
61. De La Fuente M, Seijo B, Alonso MJ. Novel hyaluronic acid-chitosan nanoparticles for ocular gene therapy. Invest Ophthalmol Vis Sci 2008;49:2016-24
62. Sharma A, Rodier J, Tandon A, et al. Attenuation of corneal myofibroblast development through nanoparticle-mediated soluble transforming growth factor-b type II receptor (sTGFbRII) gene transfer. Mol Vis 2012;18:2598-607
63. Sharma A, Tandon A, Tovey J C K, et al. Polyethelenimine-conjugated gold nanoparticles: Gene transfer potential and low toxicity in the cornea. Nanomed-nanotechnol 2011;7:505-13
64. Klausner EA, Zhang Z, Chapman RL, et al. Ultrapure chitosan oligomers as carriers for corneal gene transfer. Biomaterials 2010;31:1814-20
65. Jani PD, Singh N, Jenkins C. Nanoparticles sustain expression of Flt intraceptors in the cornea and inhibit injury-induced corneal angiogenesis. Invest Ophthalmol Vis Sci 2007;48:2030-6
66. Cai X, Conley S, Naash M. Nanoparticle applications in ocular gene therapy. Vision Res 2008;48:319-24
67. Shiraishi A, Converse RL, Liu CY, et al. Identification of the corneaspecific keratin 12 promoter by in vivo particle-mediated gene transfer. Invest Ophthalmol Vis Sci 1998;39:2554-61
68. Lu H, Lu Q, Zheng Y, Li Q. Notch signaling promotes the corneal epithelium wound healing. Mol Vis 2012;18:403-11
69. Parker DG, Brereton HM, Klebe S, et al. A steroid-inducible promoter for the cornea. Br J Ophthalmol 2009;93:1255-9
70. Beutelspacher SC, Pillai R, Watson MP. Function of indoleamine 2, 3-dioxygenase in corneal allograft rejection and prolongation of allograft survival by over-expression. Eur J Immunol 2006;36:690-700
71. Barcia RN, Dana MR, Kazlauskas A. Corneal graft rejection is accompanied by apoptosis of the endothelium and is prevented by gene therapy with bcl-xL. Am J Transplant 2007;7:2082-9
72. Fuchsluger TA, Jurkunas U, Kazlauskas A, Dana R. Corneal endothelial cells are protected from apoptosis by gene therapy. Hum Gene Ther 2011;22:549-58
73. Gong N, Pleyer U, Yang J, et al. Influence of local and systemic CTLA4Ig gene transfer on corneal allograft survival. J Gene Med 2006;8:459-67
74. Joyce NC, Harris DL, McAlister JC, et al. Overexpression of the transcription factor E2F2 induces cell cycle progression in rabbit corneal endothelial cells. Invest Ophthalmol Vis Sci 2004;45:1340-8
75. McAlister JC, Joyce NC, Harris DL, et al. Induction of replication in human corneal endothelial cells by E2F2 transcription factor cDNA transfer. Invest Ophthalmol Vis Sci 2005;46:3597-603
76. Mohan RR, Gupta R, Mehan MK, et al. Decorin transfection suppresses profibrogenic genes and myofibroblast formation in human corneal fibroblasts. Exp Eye Res 2010;91:238-45
77. Mohan RR, Tovey JC, Gupta R, et al. Decorin biology, expression, function and therapy in the cornea. Curr Mol Med 2011;11:110-28
78. Mohan RR, Tandon A, Sharma A, et al. Significant inhibition of corneal scarring in vivo with tissue-selective, targeted AAV5 decorin gene therapy. Invest Ophthalmol Vis Sci 2011;52:4833-41
79. Tandon A, Tovey JC, Sharma A, et al. Role of transforming growth factor Beta in corneal function, biology and pathology. Curr Mol Med 2010;10:565-78
80. Saika S, Ikeda K, Yamanaka O, et al. Therapeutic effects of edenoviral gene transfer of bone morphogenic protein-7 on a corneal alkali injury model in mice. Lab Invest 2005;85:474-86
81. Tandon A, Tovey J C T, Rodier JT, et al. Bone morphogenic protein 7 gene therapy inhibits fibrosis by upregulating Smad1/5/8 in rabbit cornea, in vivo. Invest Ophthalmol Vis Sci 2012;53:1086. ARVO E-abstract
82. Lai CM, Brankov M, Zaknich T, et al. Inhibition of angiogenesis by adenovirus-mediated sFlt-1 expression in a rat model of corneal neovascularization. Hum Gene Ther 2001;12:1299-310
83. Yu H, Wu J, Li H, et al. Inhibition of corneal neovascularization by recombinant adenovirus-mediated sFlk-1 expression. Biochem Biophys Res Commun 2007;361:946-52
84. Cho YK, Uehara H, Young JR, et al. Flt23k nanoparticles offer additive benefit in graft survival and anti-angiogenic effects when combined with triamcinolone. Invest Ophthalmol Vis Sci 2012;53:2328-36
85. Singh N, Amin S, Richter E, et al. Flt-1 intraceptors inhibit hypoxiainduced VEGF expression in vitro and corneal neovascularization in vivo. Invest Ophthalmol Vis Sci 2005;46:1647-52
86. Lai LJ, Xiao X, Wu JH. Inhibition of corneal neovascularization with endostatin delivered by adeno-associated viral (AAV) vector in a mouse corneal injury model. J Biomed Sci 2007;14:313-22
87. Cheng HC, Yeh SI, Tsao YP, Kuo PC. Subconjunctival injection of recombinant AAV-angiostatin ameliorates alkali burn induced corneal angiogenesis. Mol Vi 2007;13:2344-52
88. Zhou SY, Xie ZL, Xiao O, et al. Inhibition of mouse alkali burn induced-corneal neovascularization by recombinant adenovirus encoding human vasohibin-1. Mol Vi 2010;16:1389-98
89. Mohan RR, Tovey J C K, Sharma A, et al. Targeted decorin gene therapy delivered with adeno-associated virus effectively retards corneal neovascularization in vivo. PLoS One 2011;6:e26432
90. Chen S, Sun M, Meng X, et al. Pathophysiological mechanisms of autosomal dominant congenital stromal corneal dystrophy C-terminaltruncated decorin results in abnormal matrix assembly and altered expression of small leucine-rich proteoglycans. Am J Pathol 2011;179:2409-19
91. Saghizadeh M, Kramerov AA, Yu F, et al. Normalizationofwound healing and diabetic markers in organ cultured human diabetic corneas by adenoviral delivery of c-Met gene. Invest Ophthalmol Vis Sci 2010;51:1970-80
92. Thomas PB, Samant DM, Selvam S, et al. Adeno-associated virus-mediated IL-10 gene transfer suppresses lacrimal gland immunopathology in a rabbit model of autoimmune dacryoadenitis. Invest Ophthalmol Vis Sci 2010;51:5137-44
93. Selvam S, Thomas PB, Hamm-Alvarez SF, et al. Current status of gene delivery and gene therapy in lacrimal gland using viral vectors. Adv Drug Deliv Rev 2006;58:1243-57
94. Yamanaka O, Ikeda K, Saika S, et al. Gene transfer of Smad7 modulates injury-induced conjunctival wound healing in mice. Mol Vis 2006;12:841-51
95. Yamanaka O, Saika S, Ohnishi Y, et al. Inhibition of p38MAP kinase suppresses fibrogenic reaction in conjunctiva in mice. Mol Vis 2007;13:1730-9
96. Yamanaka O, Miyazaki K, Kitano A, et al. Suppression of injuryinduced conjunctiva scarring by peroxisome proliferator-activated receptor gamma gene transfer in mice. Invest Ophthalmol Vis Sci 2009;50:187-93