Advances in glaucoma treatment and management: Gene therapy

Investigative Ophthalmology and Visual Science

Volumn 53, Issue 5, 2012, Pages 2506-2510

  Borrás, Terete ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALPHA CRYSTALLIN; BONE MORPHOGENETIC PROTEIN 2; BRAIN DERIVED NEUROTROPHIC FACTOR; CYCLOOXYGENASE 2; ERYTHROPOIETIN; EYE DROPS; LENTIVIRUS VECTOR; MAGNETIC NANOPARTICLE; MATRIX METALLOPROTEINASE; NEUROTROPHIN; PARVOVIRUS VECTOR; PIGMENT EPITHELIUM DERIVED FACTOR; RHO KINASE; RHOA GUANINE NUCLEOTIDE BINDING PROTEIN; SECRETED FRIZZLED RELATED PROTEIN 1; SMALL INTERFERING RNA;

ARTICLE; CELL SURVIVAL; GENE EXPRESSION; GENE THERAPY; GLAUCOMA; HUMAN; INTRAOCULAR PRESSURE; NONHUMAN; PRIORITY JOURNAL; RETINA GANGLION CELL; SCANNING LASER OPHTHALMOSCOPE; SPECTRAL DOMAIN OPTICAL COHERENCE TOMOGRAPHY; TRABECULAR MESHWORK; VIRAL GENE DELIVERY SYSTEM; VIRUS VECTOR;

ADENOVIRIDAE; ANIMALS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLAUCOMA; HUMANS; INTRAOCULAR PRESSURE; LENTIVIRUS; MODELS, ANIMAL; NANOPARTICLES; REGULATORY ELEMENTS, TRANSCRIPTIONAL; RETINAL GANGLION CELLS; RNA, SMALL INTERFERING; TRABECULAR MESHWORK;

EID: 84860607089     PISSN: 01460404     EISSN: 15525783     Source Type: Journal    
DOI: 10.1167/iovs.12-9483o     Document Type: Article

References (33)

1. Quigley HA, Broman AT. The number of people with glaucoma worldwide in 2010 and 2020. Br J Ophthalmol. 2006;90:262-267.
2. Maguire AM, High KA, Auricchio A, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 2009;374:1597-1605.
3. Lebrun-Julien F, Di Polo A. Molecular and cell-based approaches for neuroprotection in glaucoma. Optom Vis Sci. 2008;85:417-424.
4. McKinnon SJ, Lehman DM, Tahzib NG, et al. Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model. Mol Ther. 2002;5:780-787.
5. Pease ME, Zack DJ, Berlinicke C, et al. Effect of CNTF on retinal ganglion cell survival in experimental glaucoma. Invest Ophthal-mol Vis Sci. 2009;50:2194-2200.
6. Zhou X, Li F, Kong L, Chodosh J, Cao W. Anti-inflammatory effect of pigment epithelium-derived factor in DBA/2J mice. Mol Vis. 2009;15:438-450.
7. Sullivan TA, Geisert EE, Hines-Beard J, Rex TS. Systemic adeno-associated virus-mediated gene therapy preserves retinal ganglion cells and visual function in DBA/2J glaucomatous mice. Hum Gene Ther. 2011;22:1191-1200.
8. Vittitow JL, Garg R, Rowlette LL, Epstein DL, O'Brien ET, Borras T. Gene transfer of dominant-negative RhoA increases outflow facility in perfused human anterior segment cultures. Mol Vis. 2002;8:32-44.
9. Tanihara H, Inatani M, Honjo M, Tokushige H, Azuma J, Araie M. Intraocular pressure-lowering effects and safety of topical administration of a selective ROCK inhibitor, SNJ-1656, in healthy volunteers. Arch Ophthalmol. 2008;126:309-315.
10. Xue W, Wallin R, Olmsted-Davis EA, Borras T. Matrix GLA protein function in human trabecular meshwork cells: inhibition of BMP2-induced calcification process. Invest Ophthalmol Vis Sci. 2006;47: 997-1007.
11. Wang WH, McNatt LG, Pang IH, et al. Increased expression of the WNT antagonist sFRP-1 in glaucoma elevates intraocular pressure. J Clin Invest. 2008;118:1056-1064.
12. Spiga MG, Borras T. Development of a gene therapy virus with a glucocorticoid-inducible MMP1 for the treatment of steroid glaucoma. Invest Ophthalmol Vis Sci. 2010;51:3029-3041.
13. Gerometta R, Spiga MG, Borras T, Candia OA. Treatment of sheep steroid-induced ocular hypertension with a glucocorticoid-inducible MMP1 gene therapy virus. Invest Ophthalmol Vis Sci. 2010; 51:3042-3048.
14. Tamm ER, Russell P, Johnson DH, Piatigorsky J. Human and monkey trabecular meshwork accumulate alpha B-crystallin in response to heat shock and oxidative stress. Invest Ophthalmol Vis Sci. 1996;37:2402-2413.
15. Shepard AR, Millar JC, Pang IH, Jacobson N, Wang WH, Clark AF. Adenoviral gene transfer of active human transforming growth factor-/32 elevates intraocular pressure and reduces outflow facility in rodent eyes. Invest Ophthalmol Vis Sci. 2010;51:2067-2076.
16. Balaggan KS, Ali RR. Ocular gene delivery using lentiviral vectors. Gene Ther. 2012;19:145-153
17. Miyazaki M, Ikeda Y, Yonemitsu Y, et al. Pigment epithelium-derived factor gene therapy targeting retinal ganglion cell injuries: neuroprotection against loss of function in two animal models. Hum Gene Ther. 2011;22:559-565.
18. Barraza RA, McLaren JW, Poeschla EM. Prostaglandin pathway gene therapy for sustained reduction of intraocular pressure. Mol Ther. 2010;18:491-501.
19. Raju HB, Goldberg JL. Nanotechnology for ocular therapeutics and tissue repair. Expert Rev Ophthalmol. 2008;3:431-436.
20. Comes N, Borras T. Functional delivery of synthetic naked siRNA to the human trabecular meshwork in perfused organ cultures. Mol Vis. 2007;13:1363-1374.
21. Bennett J, Ashtari M, Wellman J, et al. AAV2 Gene therapy read-ministration in three adults with congenital blindness. Sci Transl Med. 2012;4:120ra15
22. Jacobson SG, Cideciyan AV, Ratnakaram R, et al. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol. 2012;130:9-24.
23. McCarty DM, Monahan PE, Samulski RJ. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther. 2001;8:1248-1254.
24. 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;51:236-248.
25. Pasutto F, Keller KE, Weisschuh N, et al. Variants in ASB10 are associated with open-angle glaucoma. Hum Mol Genet. 2012;21: 1336-1349.
26. Fuse N. Genetic bases for glaucoma. Tohoku J Exp Med. 2010;221: 1-10.
27. Polansky JR, Kurtz RM, Fauss DJ, Kim RY, Bloom E. In vitro correlates of glucocorticoid effects on intraocular pressure. In: Krieglstein GK, ed. Glaucoma Update IV. Berlin, Heidelberg: Springer-Verlag; 1991:20-29.
28. Gong G, Kosoko-Lasaki O, Haynatzki GR, Wilson MR. Genetic dissection of myocilin glaucoma. Hum Mol Genet. 2004;13 Spec No 1:R91-R102.
29. Wiggs JL, Allingham RR, Vollrath D, et al. Prevalence of mutations in TIGR/Myocilin in patients with adult and juvenile primary open-angle glaucoma. Am J Hum Genet. 1998;63:1549-1552.
30. Alward WL, Fingert JH, Coote MA, et al. Clinical features associated with mutations in the chromosome 1 open-angle glaucoma gene (GLC1A). N Engl J Med. 1998;338:1022-1027.
31. Borras T. What is functional genomics teaching us about intraocular pressure regulation and glaucoma. In: Civan MM, ed. The Eye's Aqueous Humor. 2nd ed. San Diego: Elsevier; 2008:323-377.
32. Comes N, Borras T. Individual molecular response to elevated intraocular pressure in perfused postmortem human eyes. Physiol Genomics. 2009;38:205-225.
33. Millar JC, Clark AF, Pang IH. Assessment of aqueous humor dynamics in the mouse by a novel method of constant-flow infusion. Invest Ophthalmol Vis Sci. 2011;52:685-694.