Surgical approaches to gene and stem cell therapy for retinal disease

Human Gene Therapy

Volumn 22, Issue 5, 2011, Pages 531-535

  Stout, J Timothy ^a   Francis, Peter J ^a  

^a CASEY EYE INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

APTAMER; BEVACIZUMAB; POLY(METHYL METHACRYLATE); POLYCAPROLACTONE; POLYGLACTIN; POLYLACTIC ACID; RANIBIZUMAB; TISSUE SCAFFOLD; VASCULOTROPIN;

CANNULATION; CELL ENCAPSULATION; CELL TRANSPLANTATION; CELLULAR DISTRIBUTION; EXTRACELLULAR MATRIX; GENE THERAPY; GENETIC ASSOCIATION; HUMAN; NONHUMAN; PHOTORECEPTOR; PIGMENT EPITHELIUM; RETINA CELL; RETINA DETACHMENT; RETINA DISEASE; RETINA MACULA AGE RELATED DEGENERATION; RETINITIS PIGMENTOSA; REVIEW; STARGARDT DISEASE; STEM CELL TRANSPLANTATION; SUBRETINAL NEOVASCULARIZATION; SURGICAL APPROACH; TREATMENT OUTCOME; USHER SYNDROME; VISUAL ACUITY; VITRECTOMY; VITREOUS BODY;

CATHETERIZATION; GENE THERAPY; HUMANS; INTRAVITREAL INJECTIONS; RETINAL DISEASES; STEM CELL TRANSPLANTATION; VITRECTOMY;

EID: 79955413491     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2011.060     Document Type: Review

References (35)

1. Birch, D.G., and Liang, F.Q. (2007). Age-related macular degeneration: a target for nanotechnology derived medicines. Int. J. Nanomedicine 2, 65-77. (Pubitemid 46901783)
2. Bird, A.C., Bressler, N.M., Bressler, S.B., et al.; International ARM Epidemiological Study Group. (1995). An international classification and grading system for age-related maculopathy and age-related macular degeneration. Surv. Ophthalmol. 39, 367-374.
3. Blodi, B.A. (2004). Nutritional supplements in the prevention of age-related macular degeneration. Insight 29, 15-16; quiz 17-18. (Pubitemid 38578616)
4. Bone, R.A., Landrum, J.T., Guerra, L.H., and Ruiz, C.A. (2003). Lutein and zeaxanthin dietary supplements raise macular pigment density and serum concentrations of these caroten-oids in humans. J. Nutr. 133, 992-998. (Pubitemid 36407115)
5. Bressler, N.M. (2009). Antiangiogenic approaches to age-related macular degeneration today. Ophthalmology 116, S15-S23.
6. Bush, R.A., Lei, B., Tao, W., et al. (2004). Encapsulated cell-based intraocular delivery of ciliary neurotrophic factor in normal rabbit: Dose-dependent effects on ERG and retinal histology. Invest. Ophthalmol. Vis. Sci. 45, 2420-2430. (Pubitemid 38902618)
7. Enzmann, V., Faude, F., Wiedemann, P., and Kohen, L. (2000). The local and systemic secretion of the pro-inflammatory cy-tokine interleukin-6 after transplantation of retinal pigment epithelium cells in a rabbit model. Curr. Eye Res. 21, 530-534.
8. Enzmann, V., Hollborn, M., Kuhnhoff, S., et al. (2002). Influence of interleukin 10 and transforming growth factor-b on T cell stimulation through allogeneic retinal pigment epithelium cells in vitro. Ophthalmic Res. 34, 232-240.
9. Friedman, D.S., O'Colmain, B.J., Munoz, B., et al. (2004). Prevalence of age-related macular degeneration in the United States. Arch. Ophthalmol. 122, 564-572. (Pubitemid 38456293)
10. Gamm, D.M., Wang, S., Lu, B., et al. (2007). Protection of visual functions by human neural progenitors in a rat model of retinal disease. PLoS One 2, e338.
11. Grisanti, S., Ishioka, M., Kosiewicz, M., and Jiang, L.Q. (1997). Immunity and immune privilege elicited by cultured retinal pigment epithelial cell transplants. Invest. Ophthalmol. Vis. Sci. 38, 1619-1626. (Pubitemid 27317858)
12. Haddad, S., Chen, C.A., Santangelo, S.L., and Seddon, J.M. (2006). The genetics of age-related macular degeneration: A review of progress to date. Surv. Ophthalmol. 51, 316-363. (Pubitemid 43970736)
13. Hou, H.Y., Liang, H.L., Wang, Y.S., et al. (2010). A therapeutic strategy for choroidal neovascularization based on recruitment of mesenchymal stem cells to the sites of lesions. Mol. Ther. 18, 1837-1845.
14. Jiang, L.Q., Jorquera, M., and Streilein, J.W. (1993). Subretinal space and vitreous cavity as immunologically privileged sites for retinal allografts. Invest. Ophthalmol. Vis. Sci. 34, 3347-3354. (Pubitemid 23346667)
15. Jiang, L.Q., Jorquera, M., and Streilein, J.W. (1994). Immunologic consequences of intraocular implantation of retinal pigment epithelial allografts. Exp. Eye Res. 58, 719-728. (Pubitemid 24252920)
16. Kaiser, P.K. (2006). Antivascular endothelial growth factor agents and their development: Therapeutic implications in ocular diseases. Am. J. Ophthalmol. 142, 660-668.
17. Klein, R., Klein, B.E., Tomany, S.C., et al. (2002). Ten-year incidence and progression of age-related maculopathy: The Beaver Dam Eye Study. Ophthalmology 109, 1767-1779.
18. Klein, R., Peto, T., Bird, A., and Vannewkirk, M.R. (2004). The epidemiology of age-related macular degeneration. Am. J. Ophthalmol. 137, 486-495. (Pubitemid 38328641)
19. Maclachlan, T.K., Lukason, M., Collins, M., et al. (2011). Pre-clinical safety evaluation of AAV2-sFLT01: A gene therapy for age-related macular degeneration. Mol. Ther. 19, 326-334.
20. Redenti, S., Tao, S., Yang, J., et al. (2008). Retinal tissue engineering using mouse retinal progenitor cells and a novel biodegradable, thin-film poly(e-caprolactone) nanowire scaffold. J. Ocul. Biol. Dis. Infor. 1, 19-29.
21. Rosenfeld, P.J., Brown, D.M., Heier, J.S., et al. (2006a). Ranibi-zumab for neovascular age-related macular degeneration. N. Engl. J. Med. 355, 1419-1431. (Pubitemid 44511557)
22. Rosenfeld, P.J., Rich, R.M., and Lalwani, G.A. (2006b). Ranibi-zumab: Phase III clinical trial results. Ophthalmol. Clin. North Am. 19, 361-372. (Pubitemid 44279358)
23. Seddon, J.M., and Hennekens, C.H. (1994). Vitamins, minerals, and macular degeneration: Promising but unproven hypotheses. Arch. Ophthalmol. 112, 176-179. (Pubitemid 24058396)
24. Seiler, M.J., and Aramant, R.B. (2005). Transplantation of neu-roblastic progenitor cells as a sheet preserves and restores retinal function. Semin. Ophthalmol. 20, 31-42. (Pubitemid 40543942)
25. Seiler, M.J., Rao, B., Aramant, R.B., et al. (2010). Three-dimensional optical coherence tomography imaging of retinal sheet implants in live rats. J. Neurosci. Methods 188, 250-257.
26. Sieving, P.A., Caruso, R.C., Tao, W., et al. (2006). Ciliary neu-rotrophic factor (CNTF) for human retinal degeneration: Phase I trial of CNTF delivered by encapsulated cell intraocular implants. Proc. Natl. Acad. Sci. U.S.A. 103, 3896-3901. (Pubitemid 43376652)
27. Sodha, S., Wall, K., Redenti, S., et al. (2011). Microfabrication of a three-dimensional polycaprolactone thin-film scaffold for retinal progenitor cell encapsulation. J. Biomater. Sci. Polym. Ed. 22, 443-456.
28. Steedman, M.R., Tao, S.L., Klassen, H., and Desai, T.A. (2010). Enhanced differentiation of retinal progenitor cells using mi-crofabricated topographical cues. Biomed. Microdevices 12, 363-369.
29. Stone, E.M. (2006). A very effective treatment for neovascular macular degeneration. N. Engl. J. Med. 355, 1493-1495. (Pubitemid 44511565)
30. Sugino, I.K., Sun, Q., Wang, J., et al. (2011). Comparison of fetal RPE and human embryonic stem cell derived-RPE (hES-RPE) behavior on aged human Bruch's membrane. Invest. Oph-thalmol. Vis. Sci. (in press).
31. Tao, S., Young, C., Redenti, S., et al. (2007). Survival, migration and differentiation of retinal progenitor cells transplanted on micro-machined poly(methyl methacrylate) scaffolds to the subretinal space. Lab on a Chip 7, 695-701. (Pubitemid 46848695)
32. Tomita, M., Lavik, E., Klassen, H., et al. (2005). Biodegradable polymer composite grafts promote the survival and differentiation of retinal progenitor cells. Stem Cells 23, 1579-1588. (Pubitemid 41741007)
33. Umeda, S., Suzuki, M.T., Okamoto, H., et al. (2005). Molecular composition of drusen and possible involvement of anti-retinal autoimmunity in two different forms of macular degeneration in cynomolgus monkey (Macaca fascicularis). FASEB J. 19, 1683-1685. (Pubitemid 41429529)
34. Yao, J., Tucker, B.A., Zhang, X., et al. (2011). Robust cell integration from co-transplantation of biodegradable MMP2-PLGA microspheres with retinal progenitor cells. Biomaterials 32, 1041-1050.
35. Zhang, K., Hopkins, J.J., Heier, J.S., et al. (2011). Ciliary neuro-trophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration. Proc. Natl. Acad. Sci. U.S.A. (in press).