Advances in gene therapy for diseases of the eye

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145 · see citing works

X-linked IAP is a direct inhibitor of cell-death proteases

Deveraux, Q. L., Takahashi, R., Salvesen, G. S., & Reed, J. C. (1997) · Nature · doi:10.1038/40901 · Cited by 1,956 · see citing works

Effect of gene therapy on visual function in Leber’s congenital amaurosis

Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924 · see citing works

Pigment Epithelium-Derived Factor: A Potent Inhibitor of Angiogenesis

Dawson, D. W., Volpert, O. V., Gillis, P., Crawford, S. E., Xu, H.-J., Benedict, W., & Bouck, N. (1999) · Science · doi:10.1126/science.285.5425.245 · Cited by 1,500 · see citing works

Inhibition of vascular endothelial cell growth factor activity by an endogenously encoded soluble receptor.

Kendall, R. L., & Thomas, K. A. (1993) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.90.22.10705 · Cited by 1,393 · see citing works

The c‐IAP‐1 and c‐IAP‐2 proteins are direct inhibitors of specific caspases

Roy, N., Deveraux, Q. L., Takahashi, R., Salvesen, G. S., & Reed, J. C. (1997) · The EMBO Journal · doi:10.1093/emboj/16.23.6914 · Cited by 1,302 · see citing works

Optogenetics: 10 years of microbial opsins in neuroscience

Deisseroth, K. A. (2015) · Nature Neuroscience · doi:10.1038/nn.4091 · Cited by 1,286 · see citing works

Gene therapy restores vision in a canine model of childhood blindness

Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics · doi:10.1038/ng0501-92 · Cited by 1,202 · see citing works

A Unified Model for Apical Caspase Activation

Boatright, K. M., Renatus, M., Scott, F. L., Sperandio, S., Shin, H., Pedersen, I. M., Ricci, J., Edris, W. A., Sutherlin, D. P., Green, D. R., et al. (2003) · Molecular Cell · doi:10.1016/s1097-2765(03)00051-0 · Cited by 963 · see citing works

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.107 · Cited by 954 · see citing works

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

MacLaren, R. E., Groppe, M., Barnard, A. R., Cottriall, C. L., Tolmachova, T., Seymour, L., Clark, K. R., During, M. J., Cremers, F. P. M., Black, G. C. M., et al. (2014) · The Lancet · doi:10.1016/s0140-6736(13)62117-0 · Cited by 796 · see citing works

Ectopic Expression of a Microbial-Type Rhodopsin Restores Visual Responses in Mice with Photoreceptor Degeneration

Bi, A., Cui, J., Ma, Y.-P., Olshevskaya, E. V., Pu, M., Dizhoor, A. M., & Pan, Z. (2006) · Neuron · doi:10.1016/j.neuron.2006.02.026 · Cited by 774 · see citing works

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

Bainbridge, J. W. B., Mehat, M. S., Sundaram, V., Robbie, S. J., Barker, S. E., Ripamonti, C., Georgiadis, A., Mowat, F. M., Beattie, S. G., Gardner, P. J., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1414221 · Cited by 743 · see citing works

In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

Dalkara, D., Byrne, L. C., Klimczak, R. R., Visel, M., Yin, L., Merigan, W. H., Flannery, J. G., & Schaffer, D. V. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005708 · Cited by 734 · see citing works

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Cideciyan, A. V., Alemán, T. S., Boye, S. L., Schwartz, S., Kaushal, S., Román, A. J., Pang, J., Sumaroka, A., Windsor, E. A. M., Wilson, J. M., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0807027105 · Cited by 674 · see citing works

G Proteins and Phototransduction

Arshavsky, V. Y., Lamb, T. D., & Pugh, E. N. (2002) · Annual Review of Physiology · doi:10.1146/annurev.physiol.64.082701.102229 · Cited by 650 · see citing works

Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa

Busskamp, V., Duebel, J., Bálya, D., Fradot, M., Viney, T. J., Siegert, S., Groner, A. C., Cabuy, E., Forster, V., Seeliger, M. W., et al. (2010) · Science · doi:10.1126/science.1190897 · Cited by 639 · see citing works

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years

Jacobson, S. G. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.298 · Cited by 639 · see citing works

Ciliary neurotrophic factor (CNTF) for human retinal degeneration: Phase I trial of CNTF delivered by encapsulated cell intraocular implants

Sieving, P. A., Caruso, R. C., Tao, W., Coleman, H. R., Thompson, D. J. S., Fullmer, K. R., & Bush, R. A. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0600236103 · Cited by 591 · see citing works

Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration

Lagali, P. S., Bálya, D., Awatramani, G. B., Münch, T. A., Kim, D. S., Busskamp, V., Cepko, C. L., & Roska, B. (2008) · Nature Neuroscience · doi:10.1038/nn.2117 · Cited by 580 · see citing works

Allosteric control of an ionotropic glutamate receptor with an optical switch

Volgraf, M., Gorostiza, P., Numano, R., Krämer, R., Isacoff, E. Y., & Trauner, D. H. (2005) · Nature Chemical Biology · doi:10.1038/nchembio756 · Cited by 573 · see citing works

RPE65 is the isomerohydrolase in the retinoid visual cycle

Moiseyev, G., Chen, Y., Takahashi, Y., Wu, B. X., & Ma, J.-X. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0503460102 · Cited by 536 · see citing works

Stimulation of the insulin/mTOR pathway delays cone death in a mouse model of retinitis pigmentosa

Punzo, C., Kornacker, K., & Cepko, C. L. (2008) · Nature Neuroscience · doi:10.1038/nn.2234 · Cited by 515 · see citing works

Selective Photostimulation of Genetically ChARGed Neurons

Zemelman, B. V., Lee, G. A., Ng, M., & Miesenböck, G. (2002) · Neuron · doi:10.1016/s0896-6273(01)00574-8 · Cited by 507 · see citing works

Retinal organization in the retinal degeneration 10 (rd10) mutant mouse: A morphological and ERG study

Gargini, C., Tozzini, E. T., Mazzoni, F., & Strettoi, E. (2006) · The Journal of Comparative Neurology · doi:10.1002/cne.21144 · Cited by 496 · see citing works

Differential effect of the rd mutation on rods and cones in the mouse retina.

Carter‐Dawson, L. D., LaVail, M. M., & Sidman, R. L. (1978) · PubMed · Cited by 495 · see citing works

Protection of mouse photoreceptors by survival factors in retinal degenerations.

LaVail, M. M., Yasumura, D., Matthes, M. T., Lau-Villacorta, C., Unoki, K., Sung, C. –., & Steinberg, R. H. (1998) · PubMed · Cited by 493 · see citing works

Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness

Acland, G. M., Aguirre, G. K., Bennett, J., Alemán, T. S., Cideciyan, A. V., Bennicelli, J. L., Dejneka, N. S., Pearce-Kelling, S. E., Maguire, A. M., Palczewski, K., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.008 · Cited by 478 · see citing works

A Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis Pigmentosa

Berson, E. L. (1993) · Archives of Ophthalmology · doi:10.1001/archopht.1993.01090060049022 · Cited by 465 · see citing works

A Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis Pigmentosa

Marmor, M. F. (1993) · Archives of Ophthalmology · doi:10.1001/archopht.1993.01090110018004 · Cited by 460 · see citing works

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Cideciyan, A. V., Jacobson, S. G., Beltran, W. A., Sumaroka, A., Świder, M., Iwabe, S., Román, A. J., Olivares, M. B., Schwartz, S., Komáromy, A. M., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1218933110 · Cited by 441 · see citing works

Identification and characterization of rod-derived cone viability factor

Léveillard, T., Mohand‐Saïd, S., Lorentz, O., Hicks, D., Fintz, A.-C., Clérin, E., Simonutti, M., Forster, V., Cavusoglu, N., Chalmel, F., et al. (2004) · Nature Genetics · doi:10.1038/ng1386 · Cited by 438 · see citing works

Expression in cochlea and retina of myosin VIIa, the gene product defective in Usher syndrome type 1B.

Hasson, T., Heintzelman, M. B., Santos-Sacchi, J., Corey, D. P., & Mooseker, M. S. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.21.9815 · Cited by 427 · see citing works

Rod-Derived Cone Viability Factor Promotes Cone Survival by Stimulating Aerobic Glycolysis

Aït-Ali, N., Fridlich, R., Millet-Puel, G., Clérin, E., Delalande, F., Jaillard, C., Blond, F., Perrocheau, L., Reichman, S., Byrne, L. C., et al. (2015) · Cell · doi:10.1016/j.cell.2015.03.023 · Cited by 423 · see citing works

Antioxidants reduce cone cell death in a model of retinitis pigmentosa

Komeima, K., Rogers, B. S., Lu, L., & Campochiaro, P. A. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0604056103 · Cited by 420 · see citing works

Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle

Redmond, T. M., Poliakov, E., Yu, S., Tsai, J.-Y., Lu, Z., & Gentleman, S. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0504167102 · Cited by 409 · see citing works

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., Pierce, E. A., Chen, Y., Bennicelli, J. L., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3002865 · Cited by 395 · see citing works

Two mouse retinal degenerations caused by missense mutations in the β-subunit of rod cGMP phosphodiesterase gene

Chang, B., Hawes, N. L., Pardue, M. T., German, A. M., Hurd, R. E., Davisson, M. T., Nusinowitz, S., Rengarajan, K., Boyd, A. P., Sidney, S. S., et al. (2007) · Vision Research · doi:10.1016/j.visres.2006.11.020 · Cited by 395 · see citing works

Optochemical Genetics

Fehrentz, T., Schönberger, M., & Trauner, D. H. (2011) · Angewandte Chemie International Edition · doi:10.1002/anie.201103236 · Cited by 376 · see citing works

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

Jacobson, S. G., Cideciyan, A. V., Román, A. J., Sumaroka, A., Schwartz, S., Héon, E., & Hauswirth, W. W. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1412965 · Cited by 370 · see citing works

Pigment epithelium‐derived factor inhibits retinal and choroidal neovascularization

Mori, K., Duh, E. J., Gehlbach, P. L., Ando, A., Takahashi, K., Pearlman, J. A., Mori, K., Yang, H. S., Zack, D. J., Ettyreddy, D., et al. (2001) · Journal of Cellular Physiology · doi:10.1002/jcp.1114 · Cited by 337 · see citing works

Encapsulated cell-based delivery of CNTF reduces photoreceptor degeneration in animal models of retinitis pigmentosa.

Tao, W., Wen, R., Goddard, M. B., Sherman, S., O'Rourke, P., Stabila, P. F., Bell, W. J., Dean, B. J., Kauper, K. A., Budz, V. A., et al. (2002) · PubMed · Cited by 325 · see citing works

Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy

Bennett, J., Tanabe, T., Sun, D., Zeng, Y., Kjeldbye, H., Gouras, P., & Maguire, A. M. (1996) · Nature Medicine · doi:10.1038/nm0696-649 · Cited by 316 · see citing works

Ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration

Zhang, K., Hopkins, J. J., Heier, J. S., Birch, D. G., Halperin, L. S., Albini, T. A., Brown, D. M., Jaffe, G. J., Tao, W., & Williams, G. A. (2011) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1018987108 · Cited by 311 · see citing works

Restoration of visual function in retinal degeneration mice by ectopic expression of melanopsin

Lin, B., Koizumi, A., Tanaka, N., Panda, S., & Masland, R. H. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0806114105 · Cited by 302 · see citing works

Oxidative damage is a potential cause of cone cell death in retinitis pigmentosa

Shen, J., Yang, X., Dong, A., Petters, R. M., Peng, Y., Wong, F., & Campochiaro, P. A. (2005) · Journal of Cellular Physiology · doi:10.1002/jcp.20346 · Cited by 300 · see citing works

NRF2 promotes neuronal survival in neurodegeneration and acute nerve damage

Xiong, W., Garfinkel, A. M., Li, Y., Benowitz, L. I., & Cepko, C. L. (2015) · Journal of Clinical Investigation · doi:10.1172/jci79735 · Cited by 284 · see citing works

Longitudinal Study of Cone Photoreceptors during Retinal Degeneration and in Response to Ciliary Neurotrophic Factor Treatment

Talcott, K. E., Ratnam, K., Sundquist, S. M., Lucero, A. S., Lujan, B. J., Tao, W., Porco, T. C., Roorda, A., & Duncan, J. L. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6479 · Cited by 276 · see citing works

Virally delivered Channelrhodopsin-2 Safely and Effectively Restores Visual Function in Multiple Mouse Models of Blindness

Doroudchi, M. M., Greenberg, K. P., Liu, J., Silka, K. A., Boyden, E. S., Lockridge, J. A., Arman, A. C., Ramesh, J., Boye, S. L., Boye, S. L., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.69 · Cited by 274 · see citing works

AAV-mediated expression of CNTF promotes long-term survival and regeneration of adult rat retinal ganglion cells

Leaver, S. G., Cui, Q., Plant, G. W., Arulpragasam, A., Hisheh, S., Verhaagen, J., & Harvey, A. R. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302791 · Cited by 270 · see citing works

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Bennicelli, J., Wright, J. F., Komaromy, A., Jacobs, J. B., Hauck, B., Zelenaia, O., Mingozzi, F., Hui, D., Chung, D., Rex, T. S., et al. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300389 · Cited by 267 · see citing works

Gene therapy rescues cone function in congenital achromatopsia

Komáromy, A. M., Alexander, J. J., Rowlan, J. S., Garcia, M. M., Chiodo, V. A., Kaya, A., Tanaka, J. C., Acland, G. M., Hauswirth, W. W., & Aguirre, G. D. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq136 · Cited by 264 · see citing works

A comprehensive review of retinal gene therapy

Boye, S. E., Boye, S. L., Lewin, A. S., & Hauswirth, W. W. (2013) · Molecular Therapy · doi:10.1038/mt.2012.280 · Cited by 262 · see citing works

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

Beltran, W. A., Cideciyan, A. V., Lewin, A. S., Iwabe, S., Khanna, H., Sumaroka, A., Chiodo, V. A., Fajardo, D. S., Román, A. J., Deng, W.-T., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1118847109 · Cited by 260 · see citing works

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

Cideciyan, A. V. (2010) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2010.04.002 · Cited by 256 · see citing works

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog

Narfstro¨m, K., Katz, M. L., Bragadottir, R., Seeliger, M., Boulanger, A., Redmond, T. M., Caro, L., Lai, C.-M., & Rakoczy, P. E. (2003) · Investigative Opthalmology &amp; Visual Science · doi:10.1167/iovs.02-0595 · Cited by 252 · see citing works

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

Allocca, M., Mussolino, C., Garcia-Hoyos, M., Sanges, D., Iodice, C., Petrillo, M., Vandenberghe, L. H., Wilson, J. M., Marigo, V., Surace, E. M., et al. (2007) · Journal of Virology · doi:10.1128/jvi.01327-07 · Cited by 246 · see citing works

Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

Ghazi, N. G., Abboud, E. B., Nowilaty, S. R., Alkuraya, H., Alhommadi, A., Cai, H., Hou, R., Deng, W.-T., Boye, S. L., Almaghamsi, A., et al. (2016) · Human Genetics · doi:10.1007/s00439-016-1637-y · Cited by 243 · see citing works

Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

Vandenberghe, L. H., Bell, P., Maguire, A. M., Cearley, C. N., Xiao, R., Calcedo, R., Wang, L., Castle, M. J., Maguire, A. C., Grant, R., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3002103 · Cited by 242 · see citing works

Antioxidants slow photoreceptor cell death in mouse models of retinitis pigmentosa

Komeima, K., Rogers, B. S., & Campochiaro, P. A. (2007) · Journal of Cellular Physiology · doi:10.1002/jcp.21152 · Cited by 238 · see citing works

Optogenetic therapy for retinitis pigmentosa

Busskamp, V., Picaud, S. A., Sahel, J., & Roska, B. (2011) · Gene Therapy · doi:10.1038/gt.2011.155 · Cited by 237 · see citing works

Effect of CNTF on retinal ganglion cell survival in experimental glaucoma

Pease, M. E., Zack, D. J., Berlinicke, C., Bloom, K., Cone, F., Wang, Y., Klein, R. L., Hauswirth, W. W., & Quigley, H. A. (2009) · Investigative Opthalmology &amp; Visual Science · doi:10.1167/iovs.08-3013 · Cited by 222 · see citing works

Restoration of cone vision in a mouse model of achromatopsia

Alexander, J. J., Umino, Y., Everhart, D., Chang, B., Min, S.-H., Li, Q., Timmers, A. M., Hawes, N. L., Pang, J., Barlow, R. B., et al. (2007) · Nature Medicine · doi:10.1038/nm1596 · Cited by 218 · see citing works

Results at 2 Years after gene therapy for RPE65-deficient leber congenital amaurosis and severe early-childhood–onset retinal dystrophy

Weleber, R. G., Pennesi, M. E., Wilson, D. J., Kaushal, S., Erker, L. R., Jensen, L., McBride, M. T., Flotte, T. R., Humphries, M., Calcedo, R., et al. (2016) · Ophthalmology · doi:10.1016/j.ophtha.2016.03.003 · Cited by 218 · see citing works

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

Kong, J., Kim, S.-R., Binley, K. E., Pata, I., Doi, K., Männik, J., Zernant-Rajang, J., Kan, O., Iqball, S., Naylor, S., et al. (2008) · Gene Therapy · doi:10.1038/gt.2008.78 · Cited by 216 · see citing works

Effects of Adeno-associated Virus-vectored Ciliary Neurotrophic Factor on Retinal Structure and Function in Mice with a P216L rds/peripherin Mutation

Bok, D., Yasumura, D., Matthes, M. T., Ruíz, A., Duncan, J. L., Chappelow, A. V., Zolutukhin, S., Hauswirth, W. W., & LaVail, M. M. (2002) · Experimental Eye Research · doi:10.1006/exer.2002.1176 · Cited by 213 · see citing works

Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies

den Hollander, A. I., Black, A., Bennett, J., & Cremers, F. P. M. (2010) · Journal of Clinical Investigation · doi:10.1172/jci42258 · Cited by 209 · see citing works

Rescue from Photoreceptor Degeneration in the rd Mouse by Human Immunodeficiency Virus Vector-Mediated Gene Transfer

Takahashi, M., Miyoshi, H., Verma, I. M., & Gage, F. H. (1999) · Journal of Virology · doi:10.1128/jvi.73.9.7812-7816.1999 · Cited by 207 · see citing works

Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

Meur, G. L., Stieger, K., Smith, A. J., Weber, M., Deschamps, J., Nivard, D., Mendes-Madeira, A., Provost, N., Péréon, Y., Chérel, Y., et al. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302861 · Cited by 207 · see citing works

CNTF promotes survival of retinal ganglion cells after induction of ocular hypertension in rats: the possible involvement of STAT3 pathway

Ji, J., Elyaman, W., Yip, H. K.-F., Lee, V. W. H., Yick, L.-W., Hugon, J., & So, K. (2004) · European Journal of Neuroscience · doi:10.1111/j.0953-816x.2003.03107.x · Cited by 205 · see citing works

The Inheritance of a Retinal Abnormality in White Mice

Keeler, C. E. (1924) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.10.7.329 · Cited by 197 · see citing works

Targeting Channelrhodopsin-2 to ON-bipolar Cells With Vitreally Administered AAV Restores ON and OFF Visual Responses in Blind Mice

Macé, É., Caplette, R., Marre, O., Sengupta, A., Chaffiol, A., Barbe, P., Desrosiers, M., Bamberg, E., Sahel, J., Picaud, S. A., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.154 · Cited by 197 · see citing works

Randomized Trial of Ciliary Neurotrophic Factor Delivered by Encapsulated Cell Intraocular Implants for Retinitis Pigmentosa

Birch, D. G., Weleber, R. G., Duncan, J. L., Jaffe, G. J., & Tao, W. (2013) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2013.03.021 · Cited by 195 · see citing works

Visual Acuity after Retinal Gene Therapy for Choroideremia

Edwards, T. L., Jolly, J. K., Groppe, M., Barnard, A. R., Cottriall, C. L., Tolmachova, T., Black, G. C., Webster, A. R., Lotery, A. J., Holder, G. E., et al. (2016) · New England Journal of Medicine · doi:10.1056/nejmc1509501 · Cited by 195 · see citing works

RS-1 Gene Delivery to an Adult Rs1h Knockout Mouse Model Restores ERG b-Wave with Reversal of the Electronegative Waveform of X-Linked Retinoschisis

Zeng, Y., Takada, Y., Kjellström, S., Hiriyanna, K. T., Tanikawa, A., Wawrousek, E. F., Smaoui, N., Caruso, R. C., Bush, R. A., & Sieving, P. A. (2004) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.04-0576 · Cited by 191 · see citing works

Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial

Rakoczy, E. P., Lai, C.-M., Magno, A. L., Wikstrom, M. E., French, M. A., Pierce, C. M., Schwartz, S. D., Blumenkranz, M. S., Chalberg, T. W., Degli-Esposti, M. A., et al. (2015) · The Lancet · doi:10.1016/s0140-6736(15)00345-1 · Cited by 189 · see citing works

Restoration of Cone Vision in the CNGA3−/− Mouse Model of Congenital Complete Lack of Cone Photoreceptor Function

Michalakis, S., Mühlfriedel, R. L., Tanimoto, N., Krishnamoorthy, V., Koch, S., Fischer, M. D., Bećirović, E., Bai, L., Huber, G., Beck, S. C., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.149 · Cited by 186 · see citing works

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno‐associated virus capsid and promoter

Cronin, T., Vandenberghe, L. H., Hantz, P., Jüttner, J., Reimann, A., Kacsó, Á., Huckfeldt, R. M., Busskamp, V., Kohler, H., Lagali, P. S., et al. (2014) · EMBO Molecular Medicine · doi:10.15252/emmm.201404077 · Cited by 184 · see citing works

Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration

Byrne, L. C., Dalkara, D., Luna, G., Fisher, S. K., Clérin, E., Sahel, J., Léveillard, T., & Flannery, J. G. (2014) · Journal of Clinical Investigation · doi:10.1172/jci65654 · Cited by 182 · see citing works

LiGluR restores visual responses in rodent models of inherited blindness

Caporale, N., Kolstad, K. D., Lee, T., Tochitsky, I., Dalkara, D., Trauner, D., Kramer, R., Dan, Y., Isacoff, E. Y., & Flannery, J. G. (2011) · Molecular Therapy · doi:10.1038/mt.2011.103 · Cited by 182 · see citing works

Loss of Daylight Vision in Retinal Degeneration: Are Oxidative Stress and Metabolic Dysregulation to Blame?

Punzo, C., Xiong, W., & Cepko, C. L. (2011) · Journal of Biological Chemistry · doi:10.1074/jbc.r111.304428 · Cited by 181 · see citing works

Clinical Trial of Lutein in Patients With Retinitis Pigmentosa Receiving Vitamin A

(2010) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2010.32 · Cited by 179 · see citing works

Prolonged Recovery of Retinal Structure/Function after Gene Therapy in an Rs1h-Deficient Mouse Model of X-Linked Juvenile Retinoschisis

Min, S.-H., Molday, L. L., Seeliger, M. W., Dinculescu, A., Timmers, A. M., Janßen, A., Tonagel, F., Tanimoto, N., Weber, B. H. F., Molday, R. S., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.06.002 · Cited by 174 · see citing works

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy

Carvalho, L. S., Xu, J., Pearson, R. A., Smith, A. J., Bainbridge, J. W., Morris, L. M., Fliesler, S. J., Ding, X., & Ali, R. R. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr218 · Cited by 173 · see citing works

Functional Cone Rescue by RdCVF Protein in a Dominant Model of Retinitis Pigmentosa

Yang, Y., Mohand‐Saïd, S., Danan, A., Simonutti, M., Fontaine, V. K., Clérin, E., Picaud, S. A., Léveillard, T., & Sahel, J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.28 · Cited by 170 · see citing works

AAV-Mediated Delivery of Ciliary Neurotrophic Factor Prolongs Photoreceptor Survival in the Rhodopsin Knockout Mouse

Liang, F.-Q., Dejneka, N. S., Cohen, D. R., Krasnoperova, N. V., Lem, J., Maguire, A. M., Dudus, L., Fisher, K. J., & Bennett, J. (2001) · Molecular Therapy · doi:10.1006/mthe.2000.0252 · Cited by 167 · see citing works

Preclinical safety evaluation of AAV2-sFLT01— a gene therapy for age-related macular degeneration

MacLachlan, T. K., Lukason, M., Collins, M., Munger, R., Isenberger, E., Rogers, C., Malatos, S., DuFresne, E., Morris, J., Calcedo, R., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.258 · Cited by 166 · see citing works

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

Lai, Y., Yue, Y., & Duan, D. (2010) · Molecular Therapy · doi:10.1038/mt.2009.256 · Cited by 166 · see citing works

Baculoviral IAP Repeat-Containing-4 Protects Optic Nerve Axons in a Rat Glaucoma Model

McKinnon, S. J., Lehman, D. M., Tahzib, N. G., Ransom, N. L., Reitsamer, H. A., Liston, P., LaCasse, E. C., Li, Q., Korneluk, R. G., & Hauswirth, W. W. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0608 · Cited by 163 · see citing works

New photochemical tools for controlling neuronal activity

Krämer, R., Fortin, D. L., & Trauner, D. H. (2009) · Current Opinion in Neurobiology · doi:10.1016/j.conb.2009.09.004 · Cited by 158 · see citing works

Activated mTORC1 promotes long-term cone survival in retinitis pigmentosa mice

Venkatesh, A., Ma, S., Le, Y. Z., Hall, M. N., Rüegg, M. A., & Punzo, C. (2015) · Journal of Clinical Investigation · doi:10.1172/jci79766 · Cited by 156 · see citing works

The Status ofRPE65Gene Therapy Trials: Safety and EfficacyPreprint

Pierce, E. A., & Bennett, J. (2015) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a017285 · Cited by 151 · see citing works

Age-Related Macular Degeneration: Advances in Management and Diagnosis

Yonekawa, Y., Miller, J. W., & Kim, I. K. (2015) · Journal of Clinical Medicine · doi:10.3390/jcm4020343 · Cited by 149 · see citing works

Optogenetic Vision Restoration Using Rhodopsin for Enhanced Sensitivity

Gaub, B. M., Berry, M. H., Holt, A., Isacoff, E. Y., & Flannery, J. G. (2015) · Molecular Therapy · doi:10.1038/mt.2015.121 · Cited by 148 · see citing works

Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa

Pang, J., Dai, X., Boye, S. L., Barone, I., Boye, S. L., Mao, S., Everhart, D., Dinculescu, A., Liu, L., Umino, Y., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.273 · Cited by 148 · see citing works

Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse

Park, T. K., Wu, Z., Kjellström, S., Zeng, Y., Bush, R. A., Sieving, P. A., & Colosi, P. (2009) · Gene Therapy · doi:10.1038/gt.2009.61 · Cited by 145 · see citing works

Versatility of AAV vectors for retinal gene transfer

Surace, E. M., & Auricchio, A. (2007) · Vision Research · doi:10.1016/j.visres.2007.07.027 · Cited by 145 · see citing works

Gene Therapy for Blindness

Sahel, J., & Roska, B. (2013) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-062012-170304 · Cited by 142 · see citing works

Increased Expression of Catalase and Superoxide Dismutase 2 Reduces Cone Cell Death in Retinitis Pigmentosa

Usui, S., Komeima, K., Lee, S. Y., Jo, Y., Ueno, S., Rogers, B. S., Wu, Z., Shen, J., Lu, L., Oveson, B. C., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.47 · Cited by 141 · see citing works

AAV-Mediated Expression Targeting of Rod and Cone Photoreceptors with a Human Rhodopsin Kinase Promoter

Khani, S. C., Pawlyk, B. S., Bulgakov, O. V., Kasperek, E. M., Young, J. E., Adamian, M., Sun, X., Smith, A. J., Ali, R. R., & Li, T. (2007) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.07-0257 · Cited by 138 · see citing works

Inhibition of Retinal Neovascularization by Intraocular Viral-Mediated Delivery of Anti-angiogenic Agents

Auricchio, A., Behling, K. C., Maguire, A. M., O'Connor, E. E., Bennett, J., Wilson, J. M., & Tolentino, M. J. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0702 · Cited by 137 · see citing works

Visual Function in Mice with Photoreceptor Degeneration and Transgenic Expression of Channelrhodopsin 2 in Ganglion Cells

Thyagarajan, S., van Wyk, M., Lehmann, K., Löwel, S., Feng, G., & Wässle, H. (2010) · Journal of Neuroscience · doi:10.1523/jneurosci.4417-09.2010 · Cited by 137 · see citing works

AAV-Mediated Gene Therapy for Retinal Degeneration in the rd10 Mouse Containing a Recessive PDEβ Mutation

Pang, J., Boye, S. L., Kumar, A., Dinculescu, A., Deng, W., Li, J., Li, Q., Rani, A., Foster, T. C., Chang, B., et al. (2008) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.07-1622 · Cited by 137 · see citing works

Photoreceptor degeneration and synaptogenesis in retinal‐degenerative (rd) mice

Blanks, J. C., Adinolfi, A. M., & Lolley, R. N. (1974) · The Journal of Comparative Neurology · doi:10.1002/cne.901560108 · Cited by 136 · see citing works

Independent degeneration of photoreceptors and retinal pigment epithelium in conditional knockout mouse models of choroideremia

Tolmachova, T. (2006) · Journal of Clinical Investigation · doi:10.1172/jci26617 · Cited by 133 · see citing works

Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration

Schlichtenbrede, F. C., MacNeil, A., Bainbridge, J. W., Tschernutter, M., Thrasher, A. J., Smith, A. J., & Ali, R. R. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301929 · Cited by 132 · see citing works

Novel adeno-associated viral vectors for retinal gene therapy.

Vandenberghe, L. H., & Auricchio, A. (2012) · Gene Therapy · doi:10.1038/gt.2011.151 · Cited by 131 · see citing works

Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration

Jomary, C., Vincent, K. A., Grist, J., Neal, M., & Jones, S. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300440 · Cited by 130 · see citing works

Restoration of visual function by expression of a light-gated mammalian ion channel in retinal ganglion cells or ON-bipolar cells

Gaub, B. M., Berry, M. H., Holt, A., Reiner, A., Kienzler, M. A., Dolgova, N. V., Nikonov, S. S., Aguirre, G. D., Beltran, W. A., Flannery, J. G., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1414162111 · Cited by 129 · see citing works

Gene Replacement Therapy Rescues Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis Lacking RPGRIP

Pawlyk, B. S., Smith, A. J., Buch, P. K., Adamian, M., Hong, D.-H., Sandberg, M. A., Ali, R. R., & Li, T. (2005) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.05-0371 · Cited by 126 · see citing works

Repeated injections of a ciliary neurotrophic factor analogue leading to long-term photoreceptor survival in hereditary retinal degeneration.

Chong, V. N., Alexander, R., Waters, L., Barnett, K. C., Bird, A. C., & Luthert, P. J. (1999) · PubMed · Cited by 120 · see citing works

In Vivo Gene Therapy in Young and Adult RPE65-/- Dogs Produces Long-Term Visual Improvement

Narfström, K. L. (2003) · Journal of Heredity · doi:10.1093/jhered/esg015 · Cited by 120 · see citing works

Potential long-term inhibition of ocular neovascularisation by recombinant adeno-associated virus-mediated secretion gene therapy

Lai, Y., Shen, W., Brankov, M., Lai, C., Constable, I. J., & Rakoczy, P. E. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301695 · Cited by 120 · see citing works

Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

Tan, M. H., Smith, A. J., Pawlyk, B. S., Xu, X., Liu, X., Bainbridge, J. W., Basche, M., McIntosh, J., Tran, H. V., Nathwani, A. C., et al. (2009) · Human Molecular Genetics · doi:10.1093/hmg/ddp133 · Cited by 119 · see citing works

Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B

Hashimoto, T., Gibbs, D., Lillo, C., Azarian, S. M., Legacki, E. L., Zhang, X., Yang, X.-J., & Williams, D. S. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302897 · Cited by 118 · see citing works

The Human Rhodopsin Kinase Promoter in an AAV5 Vector Confers Rod- and Cone-Specific Expression in the Primate Retina

Boye, S. E., Alexander, J. J., Boye, S. L., Witherspoon, C. D., Sandefer, K. J., Conlon, T. J., Erger, K., Sun, J., Ryals, R., Chiodo, V. A., et al. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.125 · Cited by 117 · see citing works

Combination Angiostatin and Endostatin Gene Transfer Induces Synergistic Antiangiogenic Activity in Vitro and Antitumor Efficacy in Leukemia and Solid Tumors in Mice

Scappaticci, F. A., Smith, R., Pathak, A., Schloss, D. J., Lum, B. L., Cao, Y., Johnson, F. L., Engleman, E. G., & Nolan, G. P. (2001) · Molecular Therapy · doi:10.1006/mthe.2000.0243 · Cited by 114 · see citing works

AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia

Pang, J., Deng, W., Dai, X., Lei, B., Everhart, D., Umino, Y., Li, J., Zhang, K., Mao, S., Boye, S. L., et al. (2012) · PLoS ONE · doi:10.1371/journal.pone.0035250 · Cited by 114 · see citing works

Encapsidated adenovirus mini-chromosome-mediated delivery of genes to the retina: application to the rescue of photoreceptor degeneration

Kumar‐Singh, R., & Farber, D. B. (1998) · Human Molecular Genetics · doi:10.1093/hmg/7.12.1893 · Cited by 114 · see citing works

Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer

Ou, J., Vijayasarathy, C., Ziccardi, L., Chen, S., Zeng, Y., Marangoni, D., Pope, J. G., Bush, R. A., Wu, Z., Li, W., et al. (2015) · Journal of Clinical Investigation · doi:10.1172/jci81380 · Cited by 114 · see citing works

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

Banin, E., Gootwine, E., Obolensky, A., Ezra-Elia, R., Ejzenberg, A., Zelinger, L., Honig, H., Rosov, A., Yamin, E., Sharon, D., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.114 · Cited by 113 · see citing works

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

Mowat, F. M., Gornik, K. R., Dinculescu, A., Boye, S. L., Hauswirth, W. W., Petersen-Jones, S. M., & Bartoe, J. T. (2014) · Gene Therapy · doi:10.1038/gt.2013.64 · Cited by 108 · see citing works

Evaluation of retinal photoreceptors and pigment epithelium in a female carrier of choroideremia

Syed, N. A., Smith, J. E., John, S. K., Seabra, M. C., Aguirre, G. K., & Milam, A. H. (2001) · Ophthalmology · doi:10.1016/s0161-6420(00)00643-6 · Cited by 106 · see citing works

EIAV-Based Retinal Gene Therapy in the shaker1 Mouse Model for Usher Syndrome Type 1B: Development of UshStat

Zallocchi, M., Binley, K. M., Lad, Y., Ellis, S., Widdowson, P. S., Iqball, S., Scripps, V., Kelleher, M., Loader, J., Miskin, J., et al. (2014) · PLoS ONE · doi:10.1371/journal.pone.0094272 · Cited by 103 · see citing works

The spatio-temporal pattern of photoreceptor degeneration in the aged rd / rd mouse retina

Jimenez, A. J., García-Fernández, J. M., González, B., & Foster, R. G. (1996) · Cell and Tissue Research · doi:10.1007/s004410050579 · Cited by 100 · see citing works

Systemic but not intraocular Epo Gene Transfer Protects the Retina from Light-and Genetic-Induced Degeneration

Rex, T. S., Allocca, M., Domenici, L., Surace, E. M., Maguire, A. M., Lyubarsky, A., Cellerino, A., Bennett, J., & Auricchio, A. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.07.027 · Cited by 99 · see citing works

Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179

Nishiguchi, K. M., Carvalho, L. S., Rizzi, M., Powell, K., Holthaus, S.-M. K., Azam, S. A., Durán, Y., Ribeiro, J., Luhmann, U. F. O., Bainbridge, J. W., et al. (2015) · Nature Communications · doi:10.1038/ncomms7006 · Cited by 97 · see citing works

Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse

Boye, S. L., Boye, S. L., Pang, J., Ryals, R. C., Everhart, D., Umino, Y., Neeley, A. W., Besharse, J. C., Barlow, R. B., & Hauswirth, W. W. (2010) · PLoS ONE · doi:10.1371/journal.pone.0011306 · Cited by 95 · see citing works

Effect of Late-stage Therapy on Disease Progression in AAV-mediated Rescue of Photoreceptor Cells in the Retinoschisin-deficient Mouse

Janßen, A., Min, S.-H., Molday, L. L., Tanimoto, N., Seeliger, M. W., Hauswirth, W. W., Molday, R. S., & Weber, B. H. F. (2008) · Molecular Therapy · doi:10.1038/mt.2008.57 · Cited by 95 · see citing works

Overexpression of SOD in retina: Need for increase in H2O2-detoxifying enzyme in same cellular compartment

Usui, S., Oveson, B. C., Iwase, T., Lu, L., Lee, S. Y., Jo, Y.-J., Wu, Z., Choi, E.-Y., Samulski, R. J., & Campochiaro, P. A. (2011) · Free Radical Biology and Medicine · doi:10.1016/j.freeradbiomed.2011.06.010 · Cited by 90 · see citing works

Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

Beltran, W. A., Cideciyan, A. V., Iwabe, S., Świder, M., Kosyk, M. S., McDaid, K. S., Martynyuk, I., Ying, G., Shaffer, J., Deng, W., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1509914112 · Cited by 90 · see citing works

Periocular Gene Transfer of sFlt-1 Suppresses Ocular Neovascularization and Vascular Endothelial Growth Factor-Induced Breakdown of the Blood-Retinal Barrier

Gehlbach, P. L., Demetriades, A. M., Yamamoto, S., Deering, T. G., Xiao, W., Duh, E. J., Yang, H. S., Lai, H. C., Kovesdi, I., Carrion, M. E., et al. (2003) · Human Gene Therapy · doi:10.1089/104303403321070829 · Cited by 90 · see citing works

Regulation of Rod Phototransduction Machinery by Ciliary Neurotrophic Factor

Wen, R., Song, Y., Kjellström, S., Tanikawa, A., Liu, Y., Li, Y., Zhao, L., Bush, R. A., Laties, A. M., & Sieving, P. A. (2006) · Journal of Neuroscience · doi:10.1523/jneurosci.4021-06.2006 · Cited by 89 · see citing works

Halting progressive neurodegeneration in advanced retinitis pigmentosa

Koch, S., Tsai, Y.-T., Duong, J., Wu, W.-H., Hsu, C., Wu, W., Bonet‐Ponce, L., Lin, C., & Tsang, S. H. (2015) · Journal of Clinical Investigation · doi:10.1172/jci82462 · Cited by 85 · see citing works

Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases

Ye, G., Budzynski, E., Sonnentag, P. J., Nork, T. M., Sheibani, N., Gürel, Z., Boye, S. L., Peterson, J. J., Boye, S. L., Hauswirth, W. W., et al. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.130 · Cited by 85 · see citing works

The X-linked inhibitor of apoptosis (XIAP) prevents cell death in axotomized CNS neurons in vivo

Kügler, S., Straten, G., Kreppel, F., Isenmann, S., Liston, P., & Bähr, M. (2000) · Cell Death &amp; Differentiation · doi:10.1038/sj.cdd.4400712 · Cited by 84 · see citing works

Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia

Komáromy, A. M., Rowlan, J. S., Corr, A. T. P., Reinstein, S. L., Boye, S. L., Cooper, A. E., Gonzalez, A., Levy, B. J., Wen, R., Hauswirth, W. W., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.50 · Cited by 82 · see citing works

XIAP Protection of Photoreceptors in Animal Models of Retinitis Pigmentosa

Leonard, K. C., Petrin, D., Coupland, S. G., Baker, A. N., Leonard, B. C., LaCasse, E. C., Hauswirth, W. W., Korneluk, R. G., & Tsilfidis, C. (2007) · PLoS ONE · doi:10.1371/journal.pone.0000314 · Cited by 80 · see citing works

Rod-Cone Dysplasia (Progressive Retinal Atrophy) in Irish Setters

Aguirre, G. K., & Rubin, L. F. (1975) · Journal of the American Veterinary Medical Association · doi:10.2460/javma.1975.166.02.157 · Cited by 79 · see citing works

Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

Pawlyk, B. S., Bulgakov, O. V., Liu, X., Xu, X., Adamian, M., Sun, X., Khani, S. C., Berson, E. L., Sandberg, M. A., & Li, T. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.218 · Cited by 78 · see citing works

ω-3 Intake and Visual Acuity in Patients With Retinitis Pigmentosa Receiving Vitamin A

Berson, E. L., Rosner, B., Sandberg, M. A., Weigel-DiFranco, C., & Willett, W. C. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.2580 · Cited by 78 · see citing works

Retinal degenerations in the dog III abnormal cyclic nucleotide metabolism in rod-cone dysplasia

Aguirre, G. K., Farber, D. B., Lolley, R. N., O'Brien, P., Alligood, J. P., Fletcher, R. T., & Chader, G. J. (1982) · Experimental Eye Research · doi:10.1016/s0014-4835(82)80075-4 · Cited by 77 · see citing works

Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness

Scalabrino, M. L., Boye, S. L., Fransen, K. M., Noel, J. M., Dyka, F. M., Min, S. H., Ruan, Q., de Leeuw, C. N., Simpson, E. M., Gregg, R. G., et al. (2015) · Human Molecular Genetics · doi:10.1093/hmg/ddv341 · Cited by 75 · see citing works

Ciliary Neurotrophic Factor Protects Retinal Ganglion Cells from Secondary Cell Death During Acute Autoimmune Optic Neuritis in Rats

Maier, K., Rau, C. R., Storch, M., Sättler, M. B., Demmer, I., Weissert, R., Taheri, N., Kuhnert, A., Bähr, M., & Diem, R. (2004) · Brain Pathology · doi:10.1111/j.1750-3639.2004.tb00081.x · Cited by 74 · see citing works

Gene therapy in the second eye of RPE65-deficient dogs improves retinal function

Annear, M. J., Bartoe, J. T., Barker, S. E., Smith, A. J., Curran, P. G., Bainbridge, J. W., Ali, R. R., & Petersen‐Jones, S. M. (2010) · Gene Therapy · doi:10.1038/gt.2010.111 · Cited by 74 · see citing works

Photochemical tools to study dynamic biological processes

Specht, A., Bolze, F., Omran, Z., Nicoud, J., & Goeldner, M. P. (2009) · HFSP journal · doi:10.2976/1.3132954 · Cited by 73 · see citing works

CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa

Lipinski, D. M., Barnard, A. R., Singh, M. S., Martin, C. J., Lee, E., Davies, W. I. L., & MacLaren, R. E. (2015) · Molecular Therapy · doi:10.1038/mt.2015.68 · Cited by 73 · see citing works

Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency

Mihelec, M., Pearson, R. A., Robbie, S. J., Buch, P. K., Azam, S. A., Bainbridge, J. W., Smith, A. J., & Ali, R. R. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.069 · Cited by 72 · see citing works

Successful Gene Therapy in the RPGRIP1-deficient Dog: a Large Model of Cone–Rod Dystrophy

Lhériteau, E., Petit, L., Weber, M., Meur, G. L., Deschamps, J., Libeau, L., Mendes-Madeira, A., Guihal, C., François, A., Guyon, R., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.232 · Cited by 71 · see citing works

The melanopsin system

Benarroch, E. E. (2011) · Neurology · doi:10.1212/wnl.0b013e31821671a5 · Cited by 70 · see citing works

Lentiviral vector-mediated gene transfer in adult mouse photoreceptors is impaired by the presence of a physical barrier

Grüter, O., Kostic, C., Crippa, S. V., Perez, M.-T. R., Zografos, L., Schorderet, D. F., Munier, F. L., & Arsenijévic, Y. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302485 · Cited by 70 · see citing works

AAV-mediated transduction and targeting of retinal bipolar cells with improved mGluR6 promoters in rodents and primates

Lu, Q., Ganjawala, T. H., Ivanova, E., Cheng, J. G., Troilo, D., & Pan, Z.-H. (2016) · Gene Therapy · doi:10.1038/gt.2016.42 · Cited by 70 · see citing works

Potential Synergistic Protection of Retinal Ganglion Cells from Axotomy-Induced Apoptosis by Adenoviral Administration of Glial Cell Line-Derived Neurotrophic Factor and X-Chromosome-Linked Inhibitor of Apoptosis

Straten, G., Schmeer, C. W., Kretz, A., Gerhardt, E., Kügler, S., Schulz, J. B., Gravel, C., Bähr, M., & Isenmann, S. (2002) · Neurobiology of Disease · doi:10.1006/nbdi.2002.0543 · Cited by 68 · see citing works

Functional Rescue of Degenerating Photoreceptors in Mice Homozygous for a Hypomorphic cGMP Phosphodiesterase 6 b Allele ( Pde6b H620Q )

Davis, R. J., Tosi, J., Janisch, K. M., Kasanuki, J. M., Wang, N., Kong, J., Tsui, I. K., Cilluffo, M. C., Woodruff, M. L., Fain, G., et al. (2008) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.07-1422 · Cited by 66 · see citing works

Light-Driven Cone Arrestin Translocation in Cones of Postnatal Guanylate Cyclase-1 Knockout Mouse Retina Treated with AAV-GC1

Haire, S. E., Pang, J., Boye, S. L., Sokal, I., Craft, C. M., Palczewski, K., Hauswirth, W. W., & Semple‐Rowland, S. L. (2006) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.06-0086 · Cited by 66 · see citing works

RPE65 gene therapy slows cone loss in Rpe65-deficient dogs

Mowat, F. M., Breuwer, A. R., Bartoe, J. T., Annear, M. J., Zhang, Z., Smith, A. J., Bainbridge, J. W., Petersen‐Jones, S. M., & Ali, R. R. (2012) · Gene Therapy · doi:10.1038/gt.2012.63 · Cited by 65 · see citing works

Restoration of Vision in the pde6β-deficient Dog, a Large Animal Model of Rod-cone Dystrophy

Petit, L., Lhériteau, E., Weber, M., Meur, G. L., Deschamps, J., Provost, N., Mendes-Madeira, A., Libeau, L., Guihal, C., Colle, M.-A., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.134 · Cited by 65 · see citing works

Long-term Preservation of Cone Photoreceptors and Restoration of Cone Function by Gene Therapy in the Guanylate Cyclase-1 Knockout (GC1KO) Mouse

Boye, S. L., Conlon, T. J., Erger, K. E., Ryals, R. C., Neeley, A. W., Cossette, T. L., Pang, J., Dyka, F. M., Hauswirth, W. W., & Boye, S. L. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-7867 · Cited by 63 · see citing works

Retinal cell death and current strategies in retinal neuroprotection

Chinskey, N. D., Besirli, C. G., & Zacks, D. N. (2014) · Current Opinion in Ophthalmology · doi:10.1097/icu.0000000000000043 · Cited by 62 · see citing works

Assessment of Structure and Function Over a 3-year Period after Gene Transfer in RPE65−/− dogs

Narfström, K. L., Vaegan, Katz, M. L., Bragadóttir, R., Rakoczy, E. P., & Seeliger, M. W. (2005) · Documenta Ophthalmologica · doi:10.1007/s10633-005-3159-0 · Cited by 61 · see citing works

The Thioredoxin-like Protein Rod-derived Cone Viability Factor (RdCVFL) Interacts with TAU and Inhibits Its Phosphorylation in the Retina

Fridlich, R., Delalande, F., Jaillard, C., Lu, J., Poidevin, L., Cronin, T., Perrocheau, L., Millet-Puel, G., Niepon, M.-L., Poch, O., et al. (2009) · Molecular & Cellular Proteomics · doi:10.1074/mcp.m800406-mcp200 · Cited by 61 · see citing works

AAV-Mediated Gene Therapy in the Guanylate Cyclase (RetGC1/RetGC2) Double Knockout Mouse Model of Leber Congenital Amaurosis

Boye, S. L., Peshenko, I. V., Huang, W., Min, S.-H., McDoom, I., Kay, C. N., Liu, X., Dyka, F. M., Foster, T. C., Umino, Y., et al. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.193 · Cited by 59 · see citing works

New Diagnostic and Therapeutic Approaches for Preventing the Progression of Diabetic Retinopathy

Park, Y. G., & Roh, Y. (2015) · Journal of Diabetes Research · doi:10.1155/2016/1753584 · Cited by 58 · see citing works

Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene

Palfi, A., Millington‐Ward, S., Chadderton, N., O’Reilly, M., Goldmann, T., Humphries, M. M., Li, T., Uwe Wolfrum, B., Humphries, P., Kenna, P. F., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.119 · Cited by 56 · see citing works

Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

Wert, K. J., Davis, R. J., Sancho-Pellúz, J., Nishina, P. M., & Tsang, S. H. (2012) · Human Molecular Genetics · doi:10.1093/hmg/dds466 · Cited by 56 · see citing works

Structural and Functional Protection of Photoreceptors from MNU-Induced Retinal Degeneration by the X-Linked Inhibitor of Apoptosis

Petrin, D., Baker, A. N., Coupland, S. G., Liston, P., Narang, M. A., Damji, K. F., Leonard, B. C., Chiodo, V. A., Timmers, A. M., Hauswirth, W. W., et al. (2003) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.02-0729 · Cited by 55 · see citing works

CNGB3 -Achromatopsia Clinical Trial With CNTF: Diminished Rod Pathway Responses With No Evidence of Improvement in Cone Function

Zein, W. M., Jeffrey, B. G., Wiley, H. E., Turriff, A. E., Tumminia, S. J., Tao, W., Bush, R. A., Marangoni, D., Wen, R., Wei, L. L., et al. (2014) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.14-14860 · Cited by 54 · see citing works

Marked inhibition of retinal neovascularization in rats following soluble‐flt‐1 gene transfer

Rota, R., Riccioni, T., Zaccarini, M., Lamartina, S., Gallo, A. D., Fusco, A., Kovesdi, I., Balestrazzi, E., ABENI, D. D., Ali, R. R., et al. (2004) · The Journal of Gene Medicine · doi:10.1002/jgm.586 · Cited by 52 · see citing works

Prevention of diabetic retinopathy by intraocular soluble flt-1 gene transfer in a spontaneously diabetic rat model

Ideno, J., Mizukami, H., Kakehashi, A., Saito, Y., Okada, T., Urabe, M., Kume, A., Kuroki, M., Kawakami, M., Ishibashi, S., et al. (2007) · International Journal of Molecular Medicine · doi:10.3892/ijmm.19.1.75 · Cited by 52 · see citing works

Promising and delivering gene therapies for vision loss

Carvalho, L. S., & Vandenberghe, L. H. (2014) · Vision Research · doi:10.1016/j.visres.2014.07.013 · Cited by 49 · see citing works

AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs

Pichard, V., Provost, N., Mendes-Madeira, A., Libeau, L., Hulin, P., Tshilenge, K., Biget, M., Ameline, B., Deschamps, J., Weber, M., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.37 · Cited by 47 · see citing works

Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

Ku, C. A., Chiodo, V. A., Boye, S. L., Goldberg, A. F. X., Li, T., Hauswirth, W. W., & Ramamurthy, V. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr391 · Cited by 46 · see citing works

Gene Therapy of Inherited Retinal Degenerations: Prospects and Challenges

Trapani, I., Banfi, S., Simonelli, F., Surace, E. M., & Auricchio, A. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.030 · Cited by 46 · see citing works

CNTF gene transfer protects ganglion cells in rat retinae undergoing focal injury and branch vessel occlusion

MacLaren, R. E., Buch, P. K., Smith, A. J., Balaggan, K. S., MacNeil, A., Taylor, J. S. H., Osborne, N. N., & Ali, R. R. (2006) · Experimental Eye Research · doi:10.1016/j.exer.2006.05.019 · Cited by 45 · see citing works

IQCB1 and PDE6B Mutations Cause Similar Early Onset Retinal Degenerations in Two Closely Related Terrier Dog Breeds

Goldstein, O., Mezey, J. G., Schweitzer, P. A., Boyko, A. R., Gao, C., Bustamante, C. D., Jordan, J. A., Aguirre, G. D., & Acland, G. M. (2013) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.13-12915 · Cited by 44 · see citing works

Retinal Gene Therapy Coming of Age

Cepko, C. L., & Vandenberghe, L. H. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.050 · Cited by 43 · see citing works

AAV-Mediated Gene Replacement, Either Alone or in Combination with Physical and Pharmacological Agents, Results in Partial and Transient Protection from Photoreceptor Degeneration Associated with βPDE Deficiency

Allocca, M., Manfredi, A., Iodice, C., Vicino, U. D., & Auricchio, A. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6269 · Cited by 43 · see citing works

Combined Rod and Cone Transduction by Adeno-Associated Virus 2/8

Manfredi, A., Marrocco, E., Puppo, A., Cesi, G., Sommella, A., Corte, M. D., Rossi, S., Giunti, M., Craft, C. M., Bacci, M. L., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.154 · Cited by 43 · see citing works

Gene Therapy Fully Restores Vision to the All-Cone Nrl −/− Gucy2e −/− Mouse Model of Leber Congenital Amaurosis-1

Boye, S. L., Peterson, J. J., Choudhury, S., Min, S. H., Ruan, Q., McCullough, K. T., 张忠红, Olshevskaya, E. V., Peshenko, I. V., Hauswirth, W. W., et al. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.053 · Cited by 41 · see citing works

Inactivation of the murine X-linked juvenile retinoschisis gene, Rs1h, suggests a role of retinoschisin in retinal cell layer organization and synaptic structure

Weber, B. H. F., Schrewe, H., Molday, L. L., Gehrig, A., White, K. L., Seeliger, M. W., Jaissle, G. B., Friedburg, C., Tamm, E., & Molday, R. S. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.092528599 · Cited by 41 · see citing works

Effects on XIAP Retinal Detachment–Induced Photoreceptor Apoptosis

Zadro-Lamoureux, L. A., Zacks, D. N., Baker, A. N., Zheng, Q.-D., Hauswirth, W. W., & Tsilfidis, C. (2009) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.08-2855 · Cited by 40 · see citing works

Adeno-Associated Vector (Type 8)-Mediated Expression of Soluble Flt-1 Efficiently Inhibits Neovascularization in a Murine Choroidal Neovascularization Model

Igarashi, T., Miyake, K., Masuda, I., Takahashi, H., & Shimada, T. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.153 · Cited by 40 · see citing works

Retinal optogenetic therapies: clinical criteria for candidacy

Jacobson, S. G., Sumaroka, A., Luo, X., & Cideciyan, A. V. (2013) · Clinical Genetics · doi:10.1111/cge.12165 · Cited by 39 · see citing works

Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration

Colella, P., Iodice, C., Vicino, U. D., Annunziata, I., Surace, E. M., & Auricchio, A. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr115 · Cited by 39 · see citing works

XIAP-mediated neuroprotection in retinal ischemia

Renwick, J., Narang, M. A., Coupland, S. G., Xuan, J. Y., Baker, A. N., Brousseau, J., Petrin, D., Munger, R., Leonard, B. C., Hauswirth, W. W., et al. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302683 · Cited by 38 · see citing works

OUTCOME MEASURES AND THEIR APPLICATION IN CLINICAL TRIALS FOR RETINAL DEGENERATIVE DISEASES

Fishman, G. A., Jacobson, S. G., Alexander, K. R., Cideciyan, A. V., Birch, D. G., Weleber, R. G., & Hood, D. C. (2005) · Retina · doi:10.1097/00006982-200509000-00014 · Cited by 38 · see citing works

Systemic Gene Delivery Protects the Photoreceptors in the Retinal Degeneration Slow Mouse

Sullivan, T. A., Kodali, K., & Rex, T. S. (2010) · Neurochemical Research · doi:10.1007/s11064-010-0272-6 · Cited by 37 · see citing works

Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa

Wert, K. J., Sancho-Pellúz, J., & Tsang, S. H. (2013) · Human Molecular Genetics · doi:10.1093/hmg/ddt452 · Cited by 37 · see citing works

Gene Therapy Restores Vision and Delays Degeneration in the CNGB1−/− Mouse Model of Retinitis Pigmentosa

Michalakis, S., Koch, S., Sothilingam, V., Garrido, M. G., Tanimoto, N., Schulze, E., Bećirović, E., Koch, F., Seide, C., Beck, S. C., et al. (2014) · Advances in experimental medicine and biology · doi:10.1007/978-1-4614-3209-8_92 · Cited by 37 · see citing works

Successful Gene Therapy in Older Rpe65-Deficient Dogs Following Subretinal Injection of an Adeno-Associated Vector Expressing RPE65

Annear, M. J., Mowat, F. M., Bartoe, J. T., Querubin, J. R., Azam, S. A., Basche, M., Curran, P. G., Smith, A. J., Bainbridge, J. W., Ali, R. R., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.146 · Cited by 34 · see citing works

Proinsulin Slows Retinal Degeneration and Vision Loss in the P23H Rat Model of Retinitis Pigmentosa

Fernández‐Sánchez, L., Lax, P., Isiegas, C., Ayuso, E., Ruiz, J. M., de la Villa, P., Bosch, F., de la Rosa, E. J., & Cuenca, N. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.067 · Cited by 34 · see citing works

Improved cell metabolism prolongs photoreceptor survival upon retinal-pigmented epithelium loss in the sodium iodate induced model of geographic atrophy

Zieger, M. V., & Punzo, C. (2016) · Oncotarget · doi:10.18632/oncotarget.7330 · Cited by 32 · see citing works

AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2−/−/Cx3cr1−/− mice

Tuo, J., Pang, J., Cao, X., Shen, D., Zhang, J., Scaria, A., Wadsworth, S. C., Pechan, P. A., Boye, S. L., Hauswirth, W. W., et al. (2011) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2011.01.009 · Cited by 25 · see citing works

Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse

Palfi, A., Chadderton, N., O’Reilly, M., Nagel‐Wolfrum, K., Uwe Wolfrum, B., Bennett, J., Humphries, P., Kenna, P. F., Millington‐Ward, S., & Farrar, J. (2015) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2015.16 · Cited by 23 · see citing works

Investor Outlook: Significance of the Positive LCA2 Gene Therapy Phase III Results

Schimmer, J., & Breazzano, S. (2015) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2015.29004.sch · Cited by 21 · see citing works

Genetic deletion of S-opsin prevents rapid cone degeneration in a mouse model of Leber congenital amaurosis

Zhang, T., Enemchukwu, N. O., Jones, A. D., Wang, S., Dennis, E. M., Watt, C. B., Pugh, E. N., & Fu, Y. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu588 · Cited by 16 · see citing works

Intravitreal Ciliary Neurotrophic Factor Transiently Improves Cone-Mediated Function in a CNGB3 −/− Mouse Model of Achromatopsia

Marangoni, D., Vijayasarathy, C., Bush, R. A., Wei, L. L., Wen, R., & Sieving, P. A. (2015) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.15-16866 · Cited by 13 · see citing works

CNGB3-Achromatopsia Clinical Trial With CNTF: Diminished Rod Pathway Responses With No Evidence of Improvement in Cone Function

Langlo, C. S., Dubis, A. M., Michaelides, M., & Carroll, J. (2015) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.14-15897 · Cited by 12 · see citing works

mTORC1 sustains vision in retinitis pigmentosa

Petit, L., & Punzo, C. (2015) · Oncotarget · doi:10.18632/oncotarget.4466 · Cited by 10 · see citing works

Adeno-associated virus vector as a platform for gene therapy delivery

Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444 · see citing works

Gene Editing for <i>CEP290</i> -Associated Retinal Degeneration

Pierce, E. A., Aleman, T. S., Jayasundera, K. T., Ashimatey, B. S., Kim, K., Rashid, A., Jaskolka, M. C., Myers, R. L., Lam, B. L., Bailey, S. T., et al. (2024) · New England Journal of Medicine · doi:10.1056/nejmoa2309915 · Cited by 237 · see citing works

Advanced Formulation Approaches for Ocular Drug Delivery: State-Of-The-Art and Recent Patents

Souto, E. B., Dias-Ferreira, J., López-Machado, A., Ettcheto, M., Cano, A. C., Camins, A., Espina, M., García, M. L., & Sánchez‐López, E. (2019) · Pharmaceutics · doi:10.3390/pharmaceutics11090460 · Cited by 196 · see citing works

Aerobic Glycolysis Is Essential for Normal Rod Function and Controls Secondary Cone Death in Retinitis Pigmentosa

Petit, L., Ma, S., Cipi, J., Cheng, S.-Y., Zieger, M. V., Hay, N., & Punzo, C. (2018) · Cell Reports · doi:10.1016/j.celrep.2018.04.111 · Cited by 129 · see citing works

OPA1 gene therapy prevents retinal ganglion cell loss in a Dominant Optic Atrophy mouse model

Sarzi, E., Seveno, M., Piro-Mégy, C., Elzière, L., Quilès, M., Péquignot, M. O., Müller, A., Hamel, C., Lenaers, G., & Delettre, C. (2018) · Scientific Reports · doi:10.1038/s41598-018-20838-8 · Cited by 75 · see citing works

OPA1: How much do we know to approach therapy?

Dotto, V. D., Fogazza, M., Lenaers, G., Rugolo, M., Carelli, V., & Zanna, C. (2018) · Pharmacological Research · doi:10.1016/j.phrs.2018.02.018 · Cited by 76 · see citing works

Gene therapy for inherited retinal disease: Long-term durability of effect

Leroy, B. P., Fischer, M. D., Flannery, J. G., MacLaren, R. E., Dalkara, D., Scholl, H. P. N., Chung, D. C., Spera, C., Viriato, D., & Banhazi, J. (2023) · Ophthalmic Research · doi:10.1159/000526317 · Cited by 70 · see citing works

In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies

Yanik, M., Müller, B., Song, F., Gáll, J., Wagner, F., Wende, W., Lorenz, B., & Stieger, K. (2016) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2016.09.001 · Cited by 67 · see citing works

Gene Therapy Using a <i>miniCEP290</i> Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

Zhang, W., Li, L., Su, Q., Gao, G., & Khanna, H. (2018) · Human Gene Therapy · doi:10.1089/hum.2017.049 · Cited by 60 · see citing works

Gene therapy and the adeno‐associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations

Ramlogan‐Steel, C. A., Murali, A., Andrzejewski, S., Dhungel, B. P., Steel, J. C., & Layton, C. J. (2018) · Clinical and Experimental Ophthalmology · doi:10.1111/ceo.13416 · Cited by 59 · see citing works

Update on viral gene therapy clinical trials for retinal diseases

Cheng, S.-Y., & Punzo, C. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.159 · Cited by 49 · see citing works

Preventing Neurodegeneration by Controlling Oxidative Stress: The Role of OXR1

Volkert, M. R., & Crowley, D. J. (2020) · Frontiers in Neuroscience · doi:10.3389/fnins.2020.611904 · Cited by 48 · see citing works

Translating CRISPR-Cas Therapeutics: Approaches and Challenges

Tay, L. S., Palmer, N. D., Panwala, R., Chew, W. L., & Mali, P. (2020) · The CRISPR Journal · doi:10.1089/crispr.2020.0025 · Cited by 43 · see citing works

Novel AAV-mediated gene delivery system corrects CFTR function in pigs.

Cooney, A. L., Thornell, I., Singh, B., Shah, V. S., Stoltz, D., McCray, P., Zabner, J., & Sinn, P. (2019) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2019-0006oc · Cited by 35 · see citing works

Box Behnken design of siRNA-loaded liposomes for the treatment of a murine model of ocular keratitis caused by Acanthamoeba

Zorzi, G. K., Schuh, R. S., Maschio, V. J., Brazil, N. T., Rott, M. B., & Teixeira, H. F. (2018) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2018.10.044 · Cited by 39 · see citing works

AAV-Based Strategies for Treatment of Retinal and Choroidal Vascular Diseases: Advances in Age-Related Macular Degeneration and Diabetic Retinopathy Therapies

Castro, B. F. M., Steel, J. C., & Layton, C. J. (2023) · BioDrugs · doi:10.1007/s40259-023-00629-y · Cited by 39 · see citing works

Involvement of Innate Immune System in Late Stages of Inherited Photoreceptor Degeneration

Sudharsan, R., Beiting, D. P., Aguirre, G. D., & Beltran, W. A. (2017) · Scientific Reports · doi:10.1038/s41598-017-18236-7 · Cited by 37 · see citing works

Choroideremia

Dimopoulos, I. S., Radziwon, A., Laurent, C. D. S., & MacDonald, I. M. (2017) · Current Opinion in Ophthalmology · doi:10.1097/icu.0000000000000392 · Cited by 35 · see citing works

Design and Evaluation of Ophthalmic Delivery Formulations

Soni, V., Pandey, V., Tiwari, R., Asati, S., & Tekade, R. K. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-817909-3.00013-3 · Cited by 35 · see citing works

AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects

Mühlfriedel, R. L., Tanimoto, N., Schön, C., Sothilingam, V., Garrido, M. G., Beck, S. C., Huber, G., Biel, M., Seeliger, M. W., & Michalakis, S. (2017) · Frontiers in Neuroscience · doi:10.3389/fnins.2017.00292 · Cited by 33 · see citing works

Physiologie du signal visuel rétinien : de la phototransduction jusqu’au cycle visuel

Salesse, C. (2017) · Journal Français d Ophtalmologie · doi:10.1016/j.jfo.2016.12.006 · Cited by 32 · see citing works

Liposome-polyethylenimine complexes for the effective delivery of HuR siRNA in the treatment of diabetic retinopathy

Supe, S., Upadhya, A., Tripathi, S. D., Dighe, V. D., & Singh, K. (2023) · Drug Delivery and Translational Research · doi:10.1007/s13346-022-01281-9 · Cited by 29 · see citing works

Gene therapy for inherited retinal degenerations: initial successes and future challenges

Gupta, P. R., & Huckfeldt, R. M. (2017) · Journal of Neural Engineering · doi:10.1088/1741-2552/aa7a27 · Cited by 29 · see citing works

CRISPR/Cas9—A Promising Therapeutic Tool to Cure Blindness: Current Scenario and Future Prospects

Ahmad, I. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms231911482 · Cited by 30 · see citing works

AAV-mediated gene delivery of the calreticulin anti-angiogenic domain inhibits ocular neovascularization

Tu, L., Wang, J.-H., Barathi, V. A., Prea, S. M., He, Z., Lee, J. H., Bender, J. A., King, A. E., Logan, G. J., Alexander, I. E., et al. (2018) · Angiogenesis · doi:10.1007/s10456-017-9591-4 · Cited by 26 · see citing works

Viral-vector-delivered anti-angiogenic therapies to the eye

Koponen, S., Kokki, E., Kinnunen, K., & Ylä-Herttuala, S. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13020219 · Cited by 24 · see citing works

Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection

Petit, L., Ma, S., Cheng, S.-Y., Gao, G., & Punzo, C. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.020 · Cited by 24 · see citing works

AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery

Koponen, S., Kokki, E., Tamminen, T., & Ylä-Herttuala, S. (2023) · Frontiers in Drug Delivery · doi:10.3389/fddev.2023.1148795 · Cited by 21 · see citing works

A Destabilizing Domain Allows for Fast, Noninvasive, Conditional Control of Protein Abundance in the Mouse Eye – Implications for Ocular Gene Therapy

Datta, S., Renwick, M., Chau, V. Q., Zhang, F., Nettesheim, E. R., Lipinski, D. M., & Hulleman, J. D. (2018) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.18-24987 · Cited by 20 · see citing works

CCDC66 frameshift variant associated with a new form of early-onset progressive retinal atrophy in Portuguese Water Dogs

Murgiano, L., Becker, D., Spector, C., Carlin, K., Santana, E., Niggel, J. K., Jagannathan, V., Leeb, T., Pearce‐Kelling, S., Aguirre, G. K., et al. (2020) · Scientific Reports · doi:10.1038/s41598-020-77980-5 · Cited by 19 · see citing works

Oxidative Stress Resistance 1 Gene Therapy Retards Neurodegeneration in the rd1 Mutant Mouse Model of Retinopathy

Sahu, B., Leon, L. M., Zhang, W., Puranik, N., Periasamy, R., Khanna, H., & Volkert, M. R. (2021) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.62.12.8 · Cited by 18 · see citing works

Complex Structural PPT1 Variant Associated with Non-syndromic Canine Retinal Degeneration

Murgiano, L., Becker, D., Torjman, D., Niggel, J. K., Milano, A., Cullen, C. L., Feng, R., Wang, F., Jagannathan, V., Pearce‐Kelling, S., et al. (2018) · G3 Genes Genomes Genetics · doi:10.1534/g3.118.200859 · Cited by 16 · see citing works

Neuroprotective Effect of siRNA Entrapped in Hyaluronic Acid-Coated Lipoplexes by Intravitreal Administration

Ribeiro, M. C. S., de Miranda, M. C., Cunha, P. D. S., Andrade, G. F., Fulgêncio, G. D. O., Gomes, D. A., Fialho, S. L., Pittella, F., Charrueau, C., Escriou, V., et al. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13060845 · Cited by 16 · see citing works

Balancing gene transfection and cytotoxicity of nucleic acid carriers with focus on ocular and hepatic disorders: evaluation of hydrophobic and hydrophilic polyethyleneimine derivatives

de Oliveira, F. A., Albuquerque, L. J. C., Nascimento-Sales, M., Christoffolete, M. A., Bellettini, I. C., & Giacomelli, F. C. (2023) · Journal of Materials Chemistry B · doi:10.1039/d3tb00477e · Cited by 15 · see citing works

Low-Dose Recombinant Adeno-Associated Virus-Mediated Inhibition of Vascular Endothelial Growth Factor Can Treat Neovascular Pathologies Without Inducing Retinal Vasculitis

Cheng, S.-Y., Luo, Y., Malachi, A., Ko, J., Su, Q., Xie, J., Tian, B., Lin, H., Ke, X., Zheng, Q., et al. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.132 · Cited by 14 · see citing works

Intraocular mRNA delivery with endogenous MmPEG10-based virus-like particles

Li, M., Liu, Z., Wang, D., Ye, J., Shi, Z., Pan, C., Zhang, Q., Ju, R., Zheng, Y., & Liu, Y. (2024) · Experimental Eye Research · doi:10.1016/j.exer.2024.109899 · Cited by 14 · see citing works

Recent advancements and applications of ophthalmic gene therapy strategies: A breakthrough in ocular therapeutics

Maurya, R., Vikal, A., Narang, R. K., Patel, P., & Kurmi, B. D. (2024) · Experimental Eye Research · doi:10.1016/j.exer.2024.109983 · Cited by 13 · see citing works

Rescue of the Congenital Hereditary Endothelial Dystrophy Mouse Model by Adeno-Associated Virus–Mediated Slc4a11 Replacement

Shyam, R., Ogando, D. G., Kim, E. T., Murugan, S., Choi, M., & Bonanno, J. A. (2021) · Ophthalmology Science · doi:10.1016/j.xops.2021.100084 · Cited by 11 · see citing works

Gene therapy approaches for the treatment of retinal disorders.

Petit, L., & Punzo, C. (2016) · PubMed · Cited by 11 · see citing works

A scientometric review of hydrogel-based ocular drug delivery systems

Pal, K., Anis, A., Nayak, A. K., & Maji, S. (2021) · Elsevier eBooks · doi:10.1016/b978-0-12-820043-8.00011-6 · Cited by 11 · see citing works

Photobiomodulation promotes adenoviral gene transduction in auditory cells

Chang, S., Park, Y., Carpena, N. T., Pham, T., Chung, P., Jung, J. Y., & Lee, M. Y. (2018) · Lasers in Medical Science · doi:10.1007/s10103-018-2605-7 · Cited by 10 · see citing works

An overview of ocular drug delivery systems—conventional and novel drug delivery systems

Virmani, T., Kumar, G., Sharma, A., & Pathak, K. (2023) · Elsevier eBooks · doi:10.1016/b978-0-443-15264-1.00007-5 · Cited by 9 · see citing works

The clinical implications of molecular monitoring and analyses of inherited retinal diseases

Chacon-Camacho, O. F., García‐Montaño, L. A., & Zenteno, J. C. (2017) · Expert Review of Molecular Diagnostics · doi:10.1080/14737159.2017.1384314 · Cited by 8 · see citing works

Prospects of Optogenetic Prosthesis of the Degenerative Retina of the Eye

Оstrovsky, М. А., & Kirpichnikov, M. P. (2019) · Biochemistry (Moscow) · doi:10.1134/s0006297919050031 · Cited by 9 · see citing works

Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

Huang, D., Fletcher, S., Wilton, S. D., Palmer, N. T., McLenachan, S., Mackey, D. A., & Chen, F. K. (2017) · Vision · doi:10.3390/vision1030022 · Cited by 8 · see citing works

Twelve-month Natural History Study of Centrosomal Protein 290 (CEP290)-associated Inherited Retinal Degeneration

Pierce, E. A., Ashimatey, B. S., Jayasundera, T., Hoyng, C. B., Lam, B. L., Lorenz, B., Kim, K., Rashid, A., Myers, R., & Pennesi, M. E. (2024) · Ophthalmology Science · doi:10.1016/j.xops.2024.100483 · Cited by 7 · see citing works

Artificial vision: principles and prospects

Gilhooley, M. J., & Acheson, J. F. (2016) · Current Opinion in Neurology · doi:10.1097/wco.0000000000000412 · Cited by 7 · see citing works

Human Vascular Endothelial Growth Factor A165 Expression Induces the Mouse Model of Neovascular Age-Related Macular Degeneration

Kokki, E., Karttunen, T., Olsson, V., Kinnunen, K., & Ylä‐Herttuala, S. (2018) · Genes · doi:10.3390/genes9090438 · Cited by 7 · see citing works

Optogenetics and Vision

Kirpichnikov, M. P., & Оstrovsky, М. А. (2019) · Herald of the Russian Academy of Sciences · doi:10.1134/s1019331619010039 · Cited by 7 · see citing works

Transgene expression in mice of the Opa1 mitochondrial transmembrane protein through bicontinuous cubic lipoplexes containing gemini imidazolium surfactants

Muñoz-Úbeda, M., Semenzato, M., Franco‐Romero, A., Junquera, E., Aicart, E., Scorrano, L., & López‐Montero, I. (2021) · Journal of Nanobiotechnology · doi:10.1186/s12951-021-01167-x · Cited by 6 · see citing works

The Tumour Microenvironment and Epigenetic Regulation in BRCA1 Pathogenic Variant-Associated Breast Cancers

Tay, J. Y., Ho, J., Cheo, F. F., & Iqbal, J. (2024) · Cancers · doi:10.3390/cancers16233910 · Cited by 5 · see citing works

Nanotechnology Lighting the Way for Gene Therapy in Ophthalmopathy: From Opportunities toward Applications

Ren, W., Duan, S., Mei, C., Xie, C., Jiang, L., & Shi, Y. (2023) · Molecules · doi:10.3390/molecules28083500 · Cited by 5 · see citing works

Nucleic acid delivery to retinal cells using lipopeptides as a potential tool towards ocular gene therapies

Albuquerque, L. J. C., de Oliveira, F. A., Christoffolete, M. A., Nascimento-Sales, M., Berger, S., Wagner, E., Lächelt, U., & Giacomelli, F. C. (2023) · Journal of Colloid and Interface Science · doi:10.1016/j.jcis.2023.11.003 · Cited by 5 · see citing works

Unlocking therapeutic potential: dual gene therapy for ameliorating the disease phenotypes in a mouse model of RPE65 Leber congenital amaurosis

Liu, Y., Tai, J., Yu, C., Xu, D., Xiao, D., & Pang, J. (2024) · Frontiers in Medicine · doi:10.3389/fmed.2023.1291795 · Cited by 5 · see citing works

Frameshift Variant in AMPD2 in Cirneco dell’Etna Dogs with Retinopathy and Tremors

Murgiano, L., Niggel, J. K., Benedicenti, L., Cortellari, M., Bionda, A., Crepaldi, P., Liotta, L., Aguirre, G. K., Beltran, W. A., & Aguirre, G. D. (2024) · Genes · doi:10.3390/genes15020238 · Cited by 5 · see citing works

Gene Delivery of Calreticulin Anti-Angiogenic Domain Attenuates the Development of Choroidal Neovascularization in Rats

Bee, Y., Tu, L., Sheu, S., Lin, H.-C., Tang, J.-H., Wang, J.-H., Prea, S. M., Dusting, G. J., Wu, D., Zhong, J., et al. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.035 · Cited by 5 · see citing works

Optic nerve involvement in CACNA1F-related disease: observations from a multicentric case series

Marziali, E., den Broeck, F. V., Bargiacchi, S., Fortunato, P., Caputo, R., Sodi, A., Zaeytijd, J. D., Murro, V., Mucciolo, D. P., Giorgio, D., et al. (2022) · Ophthalmic Genetics · doi:10.1080/13816810.2022.2132514 · Cited by 4 · see citing works

Molecular Therapy for Choroideremia: Pre-clinical and Clinical Progress to Date

Kalatzis, V., Roux, A., & Meunier, I. (2021) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-021-00558-y · Cited by 3 · see citing works

High-efficiency of genetic modification using CRISPR/Cpf1 system for engineered CAR-T cell therapy

Ding, R., Chao, C., & Gao, Q. (2022) · Methods in cell biology · doi:10.1016/bs.mcb.2021.08.001 · Cited by 3 · see citing works

Optogenetic approaches to gene therapy for vision restoration in retinal degenerative diseases

Mohanty, S. K., Idigo, C., Ayyagari, A. L., & Narcisse, D. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-819291-7.00004-6 · Cited by 3 · see citing works

Translational readthrough inducing drugs for the treatment of inherited retinal dystrophies

Way, C., Cunha, D. L., & Moosajee, M. (2020) · Expert Review of Ophthalmology · doi:10.1080/17469899.2020.1762489 · Cited by 3 · see citing works

CRISPR-based Gene Editing: A Guide for the Clinician

Rossin, E. J., & Wu, D. M. (2017) · International Ophthalmology Clinics · doi:10.1097/iio.0000000000000189 · Cited by 3 · see citing works

Current trends in site and target specific delivery of nanomedicine for gene therapy

Muthuraman, A., Mehdi, S., & Rishitha, N. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-816504-1.00010-7 · Cited by 3 · see citing works

Insights into adeno-associated virus-based ocular gene therapy: A bibliometric and visual analysis

Chen, X., Yu, Y., Nie, H., Qin, X., Bai, W., Ren, J., Yao, J., Li, J., & Jiang, Q. (2023) · Medicine · doi:10.1097/md.0000000000034043 · Cited by 2 · see citing works

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models

Bower, J. J., Song, Z., & Song, L. (2022) · Journal of Visualized Experiments · doi:10.3791/63532 · Cited by 2 · see citing works

An Introduction to Providing Rehabilitation to Individuals who Have Been Implanted with the Argus II Retinal Prosthesis System

Geruschat, D. R., Rankin, M., & Dorn, J. D. (2017) · Journal of Visual Impairment & Blindness · doi:10.1177/0145482x1711100611 · Cited by 2 · see citing works

Implications of genome plasticity for drug development

López‐León, S., González‐Giraldo, Y., Ruiter-Lopez, L., & Wegman-Ostrosky, T. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-817819-5.00014-0 · Cited by 1 · see citing works

Encapsulation in the ocular system

Abdul‐Al, M., Saeinasab, M., Forouzideh, N., Keshel, S. H., Tuinea-Bobe, C. L., & Sefat, F. (2023) · Elsevier eBooks · doi:10.1016/b978-0-12-824345-9.00011-8 · Cited by 1 · see citing works

Restorative Action of Stem Cells in Neuroretinal Degenerations as a Possible Application by Ocular Mesenchymal Autograft

Limoli, P. G., Limoli, C., & Nebbioso, M. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-15717-2.00079-2 · Cited by 1 · see citing works

Novel BEST1 mutation in autosomal recessive bestrophinopathy in Japanese siblings

Yamada, R., Takagi, R., Iwamoto, S., Shimada, S., & Kakehashi, A. (2021) · Taiwan Journal of Ophthalmology · doi:10.4103/tjo.tjo_37_20 · Cited by 1 · see citing works

Extended Duration Anti-VEGF Therapy for Chorioretinal Vascular Diseases: Successes, Failures and ChallengesPreprint

Hasan, A., Miller, D. D., & Stewart, M. W. (2023) · Preprints.org · doi:10.20944/preprints202311.0789.v1 · Cited by 1 · see citing works

Genome Editing Tools und ihr Einsatz in der experimentellen Augenheilkunde

Yanik, M., Wende, W., & Stieger, K. (2017) · Klinische Monatsblätter für Augenheilkunde · doi:10.1055/s-0042-119205

Nanomedicine-Based Gene Delivery for the Retina and Posterior Segment Diseases

Lalwani, A., Shelat, P., & Patel, J. K. (2018) · Springer eBooks · doi:10.1007/978-3-319-95807-1_16

Gene therapy in animal models

Punzo, C. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-816222-4.00019-8 · Cited by 1 · see citing works

Homo creator. Von der Synthetischen Biologie zum Human Genetic Enhancement?

Boldt, J. (2020) · Technikzukünfte, Wissenschaft und Gesellschaft · doi:10.1007/978-3-658-31597-9_2

Transduction of Mouse Retina By Insect Cell Packaged Recombinant adeno-associated Viruses and Their Mutants Via Intravitreal Injection

Wei, Z., Liu, X., Li, T., Li, X., Zhou, Q., Wu, J., & Zhang, C. (2021) · Future Virology · doi:10.2217/fvl-2020-0096

The tumour microenvironment in BRCA1/BRCA2 hereditary breast cancer and the role of epigenetics in its regulationPreprint

Iqbal, J. (2023) · Qeios · doi:10.32388/0er6z3

Updates on Gene Therapy for Neovascular Age-related Macular Degeneration: A Review

Sharma, A., & Kumar, N. (2024) · EyeQuest · doi:10.4103/equest.equest_25_24

Pathophysiology and gene therapy of the optic neuropathy in Wolfram Syndrome

Jagodzinska, J. (2016) · doi:10.70675/d5ad003czb010z456bz8738za3feee533351

Caractérisation de l’environnement thymique et de la thymopoïèse en conditions physiologiques et dans le contexte de stratégies thérapeutiques par ciblage direct du thymus

Machado, A. (2021) · doi:10.70675/3a595e7ezdf5cz4f07z8550z23cafe6c4d65

Reprogramming Mammal Müller Glial Cells for Retinal Neuroregeneration

Huang, L. J., Yao, E., Yang, M., & Chen, D. F. (2026) · American Journal Of Pathology · doi:10.1016/j.ajpath.2026.06.006

Emerging therapies and innovations in the treatment of retinal degenerative diseases: A comprehensive review

Aher, P., Nikam, T. N., Laddha, U. D., Gadkari, Y. U., & Moravkar, K. K. (2026) · Next research. · doi:10.1016/j.nexres.2026.102611

Gene Therapy

Vasconcelos, H., Sallum, J. M. F., & de Carvalho, A. C. M. (2026) · Springer eBooks · doi:10.1007/978-3-032-23052-2_19

Gene therapy strategies in ophthalmology—an overview of current developments and future prospects

Krajewska, J. B., & Waszczykowska, A. (2025) · Journal of Applied Genetics · doi:10.1007/s13353-025-00973-5 · Cited by 17 · see citing works

Current Updates, Recent Trends and Future Directions of Gene Therapy on Various Eye Disorders

Rehman, S. U., Javed, U., Saleem, A., Saleem, M. H., Hameed, U., Muzaffar, K., Raza, T., Ijaz, R., & Ali, F. (2025) · Indus journal of bioscience research. · doi:10.70749/ijbr.v3i1.524 · Cited by 1 · see citing works

Republished review: Gene therapy for ocular diseases

Liu, M. M., Tuo, J., & Chan, C. (2011) · Postgraduate Medical Journal · doi:10.1136/pgmj.2009.174912rep · Cited by 15 · see citing works

Advances in Precision Therapeutics and Gene Therapy Applications for Retinal Diseases: Impact and Future Directions

AlEissa, M. M., Alhawsawi, A. A., Alonazi, R. H., Magharbil, E., Aljahdali, A., ALBalawi, H. B., Alali, N. M., Hameed, S., Abu‐Amero, K. K., & Magliyah, M. (2025) · Genes · doi:10.3390/genes16070847 · Cited by 7 · see citing works