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Volumn 84, Issue 2, 2013, Pages 175-182

Retinal optogenetic therapies: Clinical criteria for candidacy

Author keywords

Ganglion cells; Leber congenital amaurosis; Nerve fiber layer; Retinal blindness; Retinitis pigmentosa

Indexed keywords

DEHYDRODOLICHYL DIPHOSPHATE SYNTHASE; SYNTHETASE; UNCLASSIFIED DRUG;

EID: 84880171803     PISSN: 00099163     EISSN: 13990004     Source Type: Journal    
DOI: 10.1111/cge.12165     Document Type: Article
Times cited : (37)

References (44)
  • 1
    • 84873298804 scopus 로고    scopus 로고
    • From channelrhodopsins to optogenetics
    • Hegemann P, Nagel G. From channelrhodopsins to optogenetics. EMBO Mol Med 2013: 5: 173-176.
    • (2013) EMBO Mol Med , vol.5 , pp. 173-176
    • Hegemann, P.1    Nagel, G.2
  • 4
    • 84871328875 scopus 로고    scopus 로고
    • Nanomedicine for the treatment of retinal and optic nerve diseases
    • Zarbin MA, Montemagno C, Leary JF, Ritch R. Nanomedicine for the treatment of retinal and optic nerve diseases. Curr Opin Pharmacol 2013: 13: 134-148.
    • (2013) Curr Opin Pharmacol , vol.13 , pp. 134-148
    • Zarbin, M.A.1    Montemagno, C.2    Leary, J.F.3    Ritch, R.4
  • 5
    • 83455187486 scopus 로고    scopus 로고
    • Optogenetic approaches to restoring visual function in retinitis pigmentosa
    • Busskamp V, Roska B. Optogenetic approaches to restoring visual function in retinitis pigmentosa. Curr Opin Neurobiol 2011: 21: 942-946.
    • (2011) Curr Opin Neurobiol , vol.21 , pp. 942-946
    • Busskamp, V.1    Roska, B.2
  • 6
    • 77958609552 scopus 로고    scopus 로고
    • Treatment possibilities for retinitis pigmentosa
    • Jacobson SG, Cideciyan AV. Treatment possibilities for retinitis pigmentosa. N Engl J Med 2010: 363: 1669-1671.
    • (2010) N Engl J Med , vol.363 , pp. 1669-1671
    • Jacobson, S.G.1    Cideciyan, A.V.2
  • 7
    • 0038364012 scopus 로고    scopus 로고
    • Crumbs homolog 1 (CRB1) mutations result in a thick human retina with abnormal lamination
    • Jacobson SG, Cideciyan AV, Aleman TS et al. Crumbs homolog 1 (CRB1) mutations result in a thick human retina with abnormal lamination. Hum Mol Genet 2003: 12: 1073-1078.
    • (2003) Hum Mol Genet , vol.12 , pp. 1073-1078
    • Jacobson, S.G.1    Cideciyan, A.V.2    Aleman, T.S.3
  • 8
    • 20944447776 scopus 로고    scopus 로고
    • Identifying photoreceptors in blind eyes caused by RPE65 mutations: prerequisite for human gene therapy success
    • Jacobson SG, Aleman TS, Cideciyan AV et al. Identifying photoreceptors in blind eyes caused by RPE65 mutations: prerequisite for human gene therapy success. Proc Natl Acad Sci U S A 2005: 102: 6177-6182.
    • (2005) Proc Natl Acad Sci U S A , vol.102 , pp. 6177-6182
    • Jacobson, S.G.1    Aleman, T.S.2    Cideciyan, A.V.3
  • 9
    • 45549108950 scopus 로고    scopus 로고
    • Retinal laminar architecture in human retinitis pigmentosa caused by rhodopsin gene mutations
    • Aleman TS, Cideciyan AV, Sumaroka A et al. Retinal laminar architecture in human retinitis pigmentosa caused by rhodopsin gene mutations. Invest Ophthalmol Vis Sci 2008: 49: 1580-1590.
    • (2008) Invest Ophthalmol Vis Sci , vol.49 , pp. 1580-1590
    • Aleman, T.S.1    Cideciyan, A.V.2    Sumaroka, A.3
  • 10
    • 64049105380 scopus 로고    scopus 로고
    • Disease boundaries in the retina of patients with Usher syndrome caused by MYO7A gene mutations
    • Jacobson SG, Aleman TS, Sumaroka A et al. Disease boundaries in the retina of patients with Usher syndrome caused by MYO7A gene mutations. Invest Ophthalmol Vis Sci 2009: 50: 1886-1894.
    • (2009) Invest Ophthalmol Vis Sci , vol.50 , pp. 1886-1894
    • Jacobson, S.G.1    Aleman, T.S.2    Sumaroka, A.3
  • 11
    • 73349127474 scopus 로고    scopus 로고
    • CERKL mutations cause an autosomal recessive cone-rod dystrophy with inner retinopathy
    • Aleman TS, Soumittra N, Cideciyan AV et al. CERKL mutations cause an autosomal recessive cone-rod dystrophy with inner retinopathy. Invest Ophthalmol Vis Sci 2009: 50: 5944-5954.
    • (2009) Invest Ophthalmol Vis Sci , vol.50 , pp. 5944-5954
    • Aleman, T.S.1    Soumittra, N.2    Cideciyan, A.V.3
  • 12
    • 66149129178 scopus 로고    scopus 로고
    • Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate
    • Maeda T, Cideciyan AV, Maeda A et al. Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate. Hum Mol Genet 2009: 18: 2277-2287.
    • (2009) Hum Mol Genet , vol.18 , pp. 2277-2287
    • Maeda, T.1    Cideciyan, A.V.2    Maeda, A.3
  • 13
    • 79953181251 scopus 로고    scopus 로고
    • Probing mechanisms of photoreceptor degeneration in a new mouse model of the common form of autosomal dominant retinitis pigmentosa due to P23H opsin mutations
    • Sakami S, Maeda T, Bereta G et al. Probing mechanisms of photoreceptor degeneration in a new mouse model of the common form of autosomal dominant retinitis pigmentosa due to P23H opsin mutations. J Biol Chem 2011: 286: 10551-10567.
    • (2011) J Biol Chem , vol.286 , pp. 10551-10567
    • Sakami, S.1    Maeda, T.2    Bereta, G.3
  • 14
    • 80052496334 scopus 로고    scopus 로고
    • Anatomical correlates to the bands seen in the outer retina by optical coherence tomography: literature review and model
    • Spaide RF, Curcio CA. Anatomical correlates to the bands seen in the outer retina by optical coherence tomography: literature review and model. Retina 2011: 31: 1609-1619.
    • (2011) Retina , vol.31 , pp. 1609-1619
    • Spaide, R.F.1    Curcio, C.A.2
  • 15
    • 34347248079 scopus 로고    scopus 로고
    • Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation
    • Aguirre GK, Komáromy AM, Cideciyan AV et al. Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation. PLoS Med 2007: 4: e230.
    • (2007) PLoS Med , vol.4
    • Aguirre, G.K.1    Komáromy, A.M.2    Cideciyan, A.V.3
  • 16
    • 35648970061 scopus 로고    scopus 로고
    • Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosis
    • Cideciyan AV, Aleman TS, Jacobson SG et al. Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosis. Hum Mutat 2007: 28: 1074-1083.
    • (2007) Hum Mutat , vol.28 , pp. 1074-1083
    • Cideciyan, A.V.1    Aleman, T.S.2    Jacobson, S.G.3
  • 17
    • 80055100234 scopus 로고    scopus 로고
    • Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model
    • Aleman TS, Cideciyan AV, Aguirre GK et al. Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. Invest Ophthalmol Vis Sci 2011: 52: 6898-6910.
    • (2011) Invest Ophthalmol Vis Sci , vol.52 , pp. 6898-6910
    • Aleman, T.S.1    Cideciyan, A.V.2    Aguirre, G.K.3
  • 18
    • 0343550423 scopus 로고    scopus 로고
    • Morphometric study of the displacement of retinal ganglion cells subserving cones within the human fovea
    • Sjöstrand J, Popovic Z, Conradi N, Marshall J. Morphometric study of the displacement of retinal ganglion cells subserving cones within the human fovea. Graefe's Arch Clin Exp Ophthalmol 1999: 237: 1014-1023.
    • (1999) Graefe's Arch Clin Exp Ophthalmol , vol.237 , pp. 1014-1023
    • Sjöstrand, J.1    Popovic, Z.2    Conradi, N.3    Marshall, J.4
  • 19
    • 0025011140 scopus 로고
    • Topography of ganglion cells in human retina
    • Curcio CA, Allen KA. Topography of ganglion cells in human retina. J Comp Neurol 1990: 300: 5-25.
    • (1990) J Comp Neurol , vol.300 , pp. 5-25
    • Curcio, C.A.1    Allen, K.A.2
  • 20
    • 79956006692 scopus 로고    scopus 로고
    • Intravitreal injection of AAV2 transduces macaque inner retina
    • Yin L, Greenberg K, Hunter JJ et al. Intravitreal injection of AAV2 transduces macaque inner retina. Invest Ophthalmol Vis Sci 2011: 52: 2775-2783.
    • (2011) Invest Ophthalmol Vis Sci , vol.52 , pp. 2775-2783
    • Yin, L.1    Greenberg, K.2    Hunter, J.J.3
  • 21
    • 77956985453 scopus 로고    scopus 로고
    • The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders
    • Bramall AN, Wright AF, Jacobson SG, McInnes RR. The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders. Annu Rev Neurosci 2010: 33: 441-472.
    • (2010) Annu Rev Neurosci , vol.33 , pp. 441-472
    • Bramall, A.N.1    Wright, A.F.2    Jacobson, S.G.3    McInnes, R.R.4
  • 22
  • 23
    • 79851508986 scopus 로고    scopus 로고
    • A missense mutation in DHDDS, encoding dehydrodolichyl diphosphate synthase, is associated with autosomal-recessive retinitis pigmentosa in Ashkenazi Jews
    • Zelinger L, Banin E, Obolensky A et al. A missense mutation in DHDDS, encoding dehydrodolichyl diphosphate synthase, is associated with autosomal-recessive retinitis pigmentosa in Ashkenazi Jews. Am J Hum Genet 2011: 88: 207-215.
    • (2011) Am J Hum Genet , vol.88 , pp. 207-215
    • Zelinger, L.1    Banin, E.2    Obolensky, A.3
  • 24
    • 79851509221 scopus 로고    scopus 로고
    • Whole-exome sequencing links a variant in DHDDS to retinitis pigmentosa
    • Züchner S, Dallman J, Wen R et al. Whole-exome sequencing links a variant in DHDDS to retinitis pigmentosa. Am J Hum Genet 2011: 88: 201-206.
    • (2011) Am J Hum Genet , vol.88 , pp. 201-206
    • Züchner, S.1    Dallman, J.2    Wen, R.3
  • 25
    • 0027933727 scopus 로고
    • Assignment of a gene for autosomal recessive retinitis pigmentosa (RP12) to chromosome 1q31-q32.1 in an inbred and genetically heterogeneous disease population
    • van Soest S, van den Born LI, Gal A et al. Assignment of a gene for autosomal recessive retinitis pigmentosa (RP12) to chromosome 1q31-q32.1 in an inbred and genetically heterogeneous disease population. Genomics 1994: 22: 499-504.
    • (1994) Genomics , vol.22 , pp. 499-504
    • van Soest, S.1    van den Born, L.I.2    Gal, A.3
  • 26
    • 0032833350 scopus 로고    scopus 로고
    • Mutations in a human homologue of Drosophila crumbs cause retinitis pigmentosa (RP12)
    • den Hollander AI, ten Brink JB, de Kok YJ et al. Mutations in a human homologue of Drosophila crumbs cause retinitis pigmentosa (RP12). Nat Genet 1999: 23: 217-221.
    • (1999) Nat Genet , vol.23 , pp. 217-221
    • den Hollander, A.I.1    ten Brink, J.B.2    de Kok, Y.J.3
  • 27
    • 0035090259 scopus 로고    scopus 로고
    • Mutations in the CRB1 gene cause Leber congenital amaurosis
    • Lotery AJ, Jacobson SG, Fishman GA et al. Mutations in the CRB1 gene cause Leber congenital amaurosis. Arch Ophthalmol 2001: 119: 415-420.
    • (2001) Arch Ophthalmol , vol.119 , pp. 415-420
    • Lotery, A.J.1    Jacobson, S.G.2    Fishman, G.A.3
  • 28
    • 84857782236 scopus 로고    scopus 로고
    • CRB1 mutations in inherited retinal dystrophies
    • Bujakowska K, Audo I, Mohand-Saïd S et al. CRB1 mutations in inherited retinal dystrophies. Hum Mutat 2012: 33: 306-315.
    • (2012) Hum Mutat , vol.33 , pp. 306-315
    • Bujakowska, K.1    Audo, I.2    Mohand-Saïd, S.3
  • 29
    • 33749140610 scopus 로고    scopus 로고
    • Remodeling of the human retina in choroideremia: rab escort protein 1 (REP-1) mutations
    • Jacobson SG, Cideciyan AV, Sumaroka A et al. Remodeling of the human retina in choroideremia: rab escort protein 1 (REP-1) mutations. Invest Ophthalmol Vis Sci 2006: 47: 4113-4120.
    • (2006) Invest Ophthalmol Vis Sci , vol.47 , pp. 4113-4120
    • Jacobson, S.G.1    Cideciyan, A.V.2    Sumaroka, A.3
  • 30
    • 33846923884 scopus 로고    scopus 로고
    • RDH12 and RPE65, visual cycle genes causing leber congenital amaurosis, differ in disease expression
    • Jacobson SG, Cideciyan AV, Aleman TS et al. RDH12 and RPE65, visual cycle genes causing leber congenital amaurosis, differ in disease expression. Invest Ophthalmol Vis Sci 2007: 48: 332-338.
    • (2007) Invest Ophthalmol Vis Sci , vol.48 , pp. 332-338
    • Jacobson, S.G.1    Cideciyan, A.V.2    Aleman, T.S.3
  • 31
    • 35748950724 scopus 로고    scopus 로고
    • Inner retinal abnormalities in X-linked retinitis pigmentosa with RPGR mutations
    • Aleman TS, Cideciyan AV, Sumaroka A et al. Inner retinal abnormalities in X-linked retinitis pigmentosa with RPGR mutations. Invest Ophthalmol Vis Sci 2007: 48: 4759-4765.
    • (2007) Invest Ophthalmol Vis Sci , vol.48 , pp. 4759-4765
    • Aleman, T.S.1    Cideciyan, A.V.2    Sumaroka, A.3
  • 32
    • 79952578905 scopus 로고    scopus 로고
    • Cone photoreceptors are the main targets for gene therapy of NPHP5 (IQCB1) or NPHP6 (CEP290) blindness: generation of an all-cone Nphp6 hypomorph mouse that mimics the human retinal ciliopathy
    • Cideciyan AV, Rachel RA, Aleman TS et al. Cone photoreceptors are the main targets for gene therapy of NPHP5 (IQCB1) or NPHP6 (CEP290) blindness: generation of an all-cone Nphp6 hypomorph mouse that mimics the human retinal ciliopathy. Hum Mol Genet 2011: 20: 1411-1423.
    • (2011) Hum Mol Genet , vol.20 , pp. 1411-1423
    • Cideciyan, A.V.1    Rachel, R.A.2    Aleman, T.S.3
  • 33
    • 77952495178 scopus 로고    scopus 로고
    • Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis
    • Pasadhika S, Fishman GA, Stone EM et al. Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis. Invest Ophthalmol Vis Sci 2010: 5: 2608-2614.
    • (2010) Invest Ophthalmol Vis Sci , vol.5 , pp. 2608-2614
    • Pasadhika, S.1    Fishman, G.A.2    Stone, E.M.3
  • 34
    • 77954847711 scopus 로고    scopus 로고
    • Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa
    • Busskamp V, Duebel J, Balya D et al. Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science 2010: 329: 413-417.
    • (2010) Science , vol.329 , pp. 413-417
    • Busskamp, V.1    Duebel, J.2    Balya, D.3
  • 35
    • 77954620055 scopus 로고    scopus 로고
    • Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy
    • Cideciyan AV. Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy. Prog Retin Eye Res 2010: 29: 398-427.
    • (2010) Prog Retin Eye Res , vol.29 , pp. 398-427
    • Cideciyan, A.V.1
  • 36
    • 84873453664 scopus 로고    scopus 로고
    • Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement
    • Cideciyan AV, Jacobson SG, Beltran WA et al. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci U S A 2013: 110: E517-E525.
    • (2013) Proc Natl Acad Sci U S A , vol.110
    • Cideciyan, A.V.1    Jacobson, S.G.2    Beltran, W.A.3
  • 37
    • 84859269427 scopus 로고    scopus 로고
    • Interim results from the international trial of Second Sight's visual prosthesis
    • Humayun MS, Dorn JD, da Cruz L et al. Interim results from the international trial of Second Sight's visual prosthesis. Ophthalmology 2012: 119: 779-788.
    • (2012) Ophthalmology , vol.119 , pp. 779-788
    • Humayun, M.S.1    Dorn, J.D.2    da Cruz, L.3
  • 38
    • 0026460822 scopus 로고
    • Morphometric analysis of macular photoreceptors and ganglion cells in retinas with retinitis pigmentosa
    • Stone JL, Barlow WE, Humayun MS, de Juan E Jr, Milam AH. Morphometric analysis of macular photoreceptors and ganglion cells in retinas with retinitis pigmentosa. Arch Ophthalmol 1992: 110: 1634-1639.
    • (1992) Arch Ophthalmol , vol.110 , pp. 1634-1639
    • Stone, J.L.1    Barlow, W.E.2    Humayun, M.S.3    de Juan Jr., E.4    Milam, A.H.5
  • 39
    • 0030940097 scopus 로고    scopus 로고
    • Preservation of the inner retina in retinitis pigmentosa. A morphometric analysis
    • Santos A, Humayun MS, de Juan E Jr et al. Preservation of the inner retina in retinitis pigmentosa. A morphometric analysis. Arch Ophthalmol 1997: 115: 511-515.
    • (1997) Arch Ophthalmol , vol.115 , pp. 511-515
    • Santos, A.1    Humayun, M.S.2    de Juan Jr., E.3
  • 40
    • 0032952549 scopus 로고    scopus 로고
    • Morphometric analysis of the extramacular retina from postmortem eyes with retinitis pigmentosa
    • Humayun MS, Prince M, de Juan E Jr et al. Morphometric analysis of the extramacular retina from postmortem eyes with retinitis pigmentosa. Invest Ophthalmol Vis Sci 1999: 40: 143-148.
    • (1999) Invest Ophthalmol Vis Sci , vol.40 , pp. 143-148
    • Humayun, M.S.1    Prince, M.2    de Juan Jr., E.3
  • 42
    • 33749061496 scopus 로고    scopus 로고
    • Evaluation of residual retinal function by pupillary constrictions and phosphenes using transcorneal electrical stimulation in patients with retinal degeneration
    • Morimoto T, Fukui T, Matsushita K et al. Evaluation of residual retinal function by pupillary constrictions and phosphenes using transcorneal electrical stimulation in patients with retinal degeneration. Graefes Arch Clin Exp Ophthalmol 2006: 244: 1283-1292.
    • (2006) Graefes Arch Clin Exp Ophthalmol , vol.244 , pp. 1283-1292
    • Morimoto, T.1    Fukui, T.2    Matsushita, K.3
  • 43
    • 84868326123 scopus 로고    scopus 로고
    • Phosphene thresholds elicited by transcorneal electrical stimulation in healthy subjects and patients with retinal diseases
    • Naycheva L, Schatz A, Röck T et al. Phosphene thresholds elicited by transcorneal electrical stimulation in healthy subjects and patients with retinal diseases. Invest Ophthalmol Vis Sci 2012: 53: 7440-7448.
    • (2012) Invest Ophthalmol Vis Sci , vol.53 , pp. 7440-7448
    • Naycheva, L.1    Schatz, A.2    Röck, T.3
  • 44
    • 84861819775 scopus 로고    scopus 로고
    • A review and update on the current status of stem cell therapy and the retina
    • Ong JM, da Cruz L. A review and update on the current status of stem cell therapy and the retina. Br Med Bull 2012: 102: 133-146.
    • (2012) Br Med Bull , vol.102 , pp. 133-146
    • Ong, J.M.1    da Cruz, L.2


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