Translational research in retinology

Clinical Ophthalmology

Volumn 5, Issue 1, 2011, Pages 1493-1498

  Siqueira, Rubens Camargo ^a,b   Jorge, Rodrigo ^b  

^a Rubens Siqueira Research Center   (Brazil)

^b UNIVERSITY OF SÃO PAULO   (Brazil)

Author keywords

Gene therapy; Retinal diseases; Stem cell; Translational

Indexed keywords

ALTERNATIVE COMPLEMENT PATHWAY C3 C5 CONVERTASE; BEVACIZUMAB; COMPLEMENT COMPONENT C3; COMPLEMENT FACTOR H; COMPLEMENT FACTOR I; FENRETINIDE; GROWTH FACTOR; ISOMERASE; LIPOFUSCIN; NEUROPROTECTIVE AGENT; NEUROTROPHIC FACTOR; PARVOVIRUS VECTOR; RANIBIZUMAB; RETINAL PIGMENT EPITHELIUM PROTEIN 65; UNCLASSIFIED DRUG; VASCULOTROPIN;

ABCA4 GENE; ARTICLE; AUTOLOGOUS BONE MARROW TRANSPLANTATION; CONGENITAL CATARACT; CORNEA DYSTROPHY; DRUG EFFECT; ELECTRONIC MEDICAL RECORD; GENE; GENE REPLACEMENT THERAPY; GENE THERAPY; GENETIC SCREENING; GLAUCOMA; HUMAN; LEBER CONGENITAL AMAUROSIS; NONHUMAN; OPHTHALMOLOGY; OPTIC NERVE DISEASE; PERSONALIZED MEDICINE; PROSTHESIS; RETINA DISEASE; RETINA MACULA AGE RELATED DEGENERATION; RETINA MACULA DEGENERATION; RETINITIS PIGMENTOSA; STARGARDT DISEASE; STEM CELL TRANSPLANTATION; SUBRETINAL NEOVASCULARIZATION; TRANSLATIONAL RESEARCH; VITREORETINOPATHY;

EID: 81155139145     PISSN: 11775467     EISSN: 11775483     Source Type: Journal    
DOI: 10.2147/opth.s25249     Document Type: Article

References (42)

1. Franco de Oliveira LV. From bench to bedside. ConScientiae Saúde. 2009;8:545-547.
2. Lean MEJ, Mann JI, Hoek JA, Elliot RM, Schofield G. Translational Research: from evidence-based medicine to sustainable solutions for public health problems. Br Med J. 2008;337:a863.
3. Hiss RG. Fundamental issues in translational research. Translational research - two phases of a continuum. From clinical trials to community: the science of translating diabetes and obesity research. Natcher Conference Center, National Institutes of Health, Bethesda, MD. Available at: http://www2.niddk.nih.gov/Research/Reports/ConferenceReports/2004/diabetes_translation.htm. Accessed on October 4, 2011. 2004:11-14.
4. Demeure MJ. Personalized medicine: the future is not what it used to be. Surgery. 2009;146:971-978.
5. Hamburg MA, Collins FS. The path to personalized medicine. N Engl J Med. 2010;363:301-304.
6. Swaroop A, Chew EY, Rickman CB, Abecasis GR. Unraveling a multifactorial late-onset disease: from genetic susceptibility to disease mechanisms for age-related macular degeneration. Annu Rev Genomics Hum Genet. 2009;10:19-43.
7. Schindler EI, Nylen EL, Ko AC, et al. Deducing the pathogenic contribution of recessive ABCA4 alleles in an outbred population. Hum Mol Genet. 2010;19:3693-3701.
8. Cideciyan AV, Swider M, Aleman TS, et al. ABCA4 disease progression and a proposed strategy for gene therapy. Hum Mol Genet. 2009;18:931-941.
9. Sheffield VC, Stone EM. Genomics and the eye. N Engl J Med. 2011; 364:1932-1942.
10. Weng J, Mata NL, Azarian SM, Tzekov RT, Birch DG, Travis GH. Insights into the function of Rim protein in photoreceptors and etiology of Stargardt's disease from the phenotype in abcr knockout mice. Cell. 1999;98:13-23.
11. Radu RA, Han Y, Bui TV, et al. Reductions in serum vitamin A arrest accumulation of toxic retinal fluorophores: a potential therapy for treatment of lipofuscin-based retinal diseases. Invest Ophthalmol Vis Sci. 2005;46: 4393-4401. [Erratum, Invest Ophthalmol Vis Sci. 2006;47:3735.]
12. Sieving PA, Caruso RC, Tao W, et al. Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants. Proc Natl Acad Sci U S A. 2006;103:3896-3901.
13. Boatright JH, Moring AG, McElroy C, et al. Tool from ancient pharmacopoeia prevents vision loss. Mol Vis. 2006;12:1706-1714.
14. den Hollander AI, Black A, Bennett J, Cremers FP. Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. J Clin Invest. September 1, 2010;120(9):3042-3053. [Erratum, J Clin Invest. 2011;121:456-457.]
15. Acland GM, Aguirre GD, Ray J, et al. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet. 2001;28:92-95.
16. Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008;358:2240-2248.
17. Bainbridge JW, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008;358:2231-2239.
18. Hauswirth WW, Aleman TS, Kaushal S, et al. Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther. 2008;19:979-990.
19. Chen W, Stambolian D, Edwards AO, et al. Genetic variants near TIMP3 and high-density lipoprotein-associated loci influence susceptibility to age-related macular degeneration. Proc Natl Acad Sci U S A. 2010;107:7401-7406.
20. Awh CC, Zanke B. Applying genetic risk data to clinical practice. Retina Today. 2011:78-79.
21. Haines JL, Spencer KM, Pericak-Vance MA. Bringing the genetics of macular degeneration into focus. Proc Natl Acad Sci U S A. 2007;104:16725-16726.
22. Klein RJ, Zeiss C, Chew EY, et al. Complement factor H polymorphism in age-related macular degeneration. Science. 2005;308:385-389.
23. Fagerness JA, Maller JB, Neale BM, Reynolds RC, Daly MJ, Seddon JM. Variation near complement factor I is associated with risk of advanced AMD. Eur J Hum Genet. 2009;17:100-104.
24. Yates JR, Sepp T, Matharu BK, et al. Complement C3 variant and the risk of age-related macular degeneration. N Engl J Med. 2007;357:553-561.
25. Jakobsdottir J, Conley YP, Weeks DE, Ferrell RE, Gorin MB. C2 and CFB genes in age-related maculopathy and joint action with CFH and LOC387715 genes. PLoS ONE. 2008;3:2199.
26. Siqueira RC, Voltarelli JC, Messias AM, Jorge R. Possible mechanisms of retinal function recovery with the use of cell therapy with bone marrow-derived stem cells. Arq Bras Oftalmol. 2010;73:474-479.
27. Lanza R, Rosenthal N. The stem cell challenge. Sci Am. 2004;290: 93-99.
28. Machaliñski A, Baumert B, Kuprjanowicz L, Wiszniewska B, Karczewicz D, Machaliñski B. Potential application of adult stem cells in retinal repair-challenge for regenerative medicine. Curr Eye Res. 2009;34:748-760.
29. Otani A, Dorrell MI, Kinder K, et al. Rescue of retinal degeneration by intravitreally injected adult bone marrow-derived lineage negative hematopoietic stem cells. J Clin Invest. 2004;114:765-774. Comment in: J Clin Invest. 2004;114:755-757.
30. Dahlmann-Noor A, Vijay S, Jayaram H, Limb A, Khaw PT. Current approaches and future prospects for stem cell rescue and regeneration of the retina and optic nerve. Can J Ophthalmol. 2010;45:333-341.
31. Jonas JB, Witzens-Harig M, Arseniev L, Ho AD. Intravitreal autologous bone marrow derived mononuclear cell transplantation: a feasibility report. Acta Ophthalmol. 2008;86:225-226.
32. Jonas JB, Witzens-Harig M, Arseniev L, Ho AD. Intravitreal autologous bone marrow-derived mononuclear cell transplantation. Acta Ophthalmol. 2010;88:131-132.
33. Siqueira RC, Messias A, Voltarelli JC, Scott IU, Jorge R. Intravitreal injection of autologous bone marrow-derived mononuclear cells for hereditary retinal dystrophy: a phase I trial. Retina. 2011; 31:1207-1214.
34. Schwartz S. Sub-retinal Transplantation of hESC Derived RPE (MA09-hRPE) Cells in Patients with Stargardt's Macular Dystrophy. NCT01345006. Clinicaltrials.gov.
35. Dowling J. Current and future prospects for optoelectronic retinal prostheses. Eye (Lond). 2009;23:1999-2005.
36. Ong JM, Cruz LD. The bionic eye: a review. Clin Exp Ophthalmol. 2011. [Epub ahead of print.]
37. Humayun MS, Dorn JD, Ahuja AK, et al. Preliminary 6 month results from the Argus II epiretinal prosthesis feasibility study. Conf Proc IEEE Eng Med Biol Soc. 2009;2009:4566-4568.
38. Gekeler F, Kopp A, Sachs H, et al. Visualisation of active subretinal implants with external connections by high-resolution CT. Br J Ophthalmol. 2010;94:843-847.
39. Petermeier K, Frank C, Gekeler F, Spitzer MS, Messias A, Szurman P. Influence of the pupil size on visual quality and spherical aberration after implantation of the Tecnis 1-piece intraocular lens. Br J Ophthalmol. 2011;95:42-45.
40. Besch D, Sachs H, Szurman P, et al. Extraocular surgery for implantation of an active subretinal visual prosthesis with external connections: feasibility and outcome in seven patients. Br J Ophthalmol. 2008;92:1361-1368.
41. Chader G, Weiland J, Humayun M. Artificial vision: needs, functioning, and testing of a retinal electronic prosthesis. Prog Brain Res. 2009;175:317-332.
42. Finlayson P, Iezzi R. Glutamate stimulation of retinal ganglion cells in normal and s334ter rat retinas: a candidate for a neurotransmitterbased retinal prosthesis. Invest Ophthalmol Vis Sci. 2010;51: 3619-3628.