Helper-Dependent Adenovirus for the Gene Therapy of Proliferative Retinopathies: Stable Gene Transfer, Regulated Gene Expression and Therapeutic Efficacy

Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method

Livak, K. J., & Schmittgen, T. D. (2001) · Methods · doi:10.1006/meth.2001.1262 · Cited by 180,860 · see citing works

The biology of VEGF and its receptors

Ferrara, N., Gerber, H.-P., & LeCouter, J. (2003) · Nature Medicine · doi:10.1038/nm0603-669 · Cited by 9,591 · see citing works

Diabetic Retinopathy

Lang, G. E., Vaiser, A., Snyder, W. B., & Lynn, J. R. (1974) · doi:10.1159/isbn.978-3-318-01439-6 · Cited by 5,583 · see citing works

Pegaptanib for neovascular age-related macular degeneration

Gragoudas, E. S., Adamis, A. P., Cunningham, E. T., Feinsod, M., & Guyer, D. R. (2004) · New England Journal of Medicine · doi:10.1056/nejmoa042760 · Cited by 2,337 · see citing works

Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease

Ng, E. W. M., Shima, D. T., Calias, P., Cunningham, E. T., Guyer, D. R., & Adamis, A. P. (2006) · Nature Reviews Drug Discovery · doi:10.1038/nrd1955 · Cited by 1,591 · 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

Suppression of retinal neovascularization in vivo by inhibition of vascular endothelial growth factor (VEGF) using soluble VEGF-receptor chimeric proteins.

Aiello, L. P., Pierce, E. A., Foley, E. D., Takagi, H., Chen, H., Riddle, L. M., Ferrara, N., King, G. L., & Smith, L. E. H. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.23.10457 · Cited by 1,258 · see citing works

Vascular endothelial growth factor/vascular permeability factor expression in a mouse model of retinal neovascularization.

Pierce, E. A., Avery, R. L., Foley, E. D., Aiello, L. P., & Smith, L. E. H. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.3.905 · Cited by 1,026 · see citing works

Diabetic Retinopathy

Frank, R. N. (2003) · New England Journal of Medicine · doi:10.1056/nejmra021678 · Cited by 891 · see citing works

The Müller cell: a functional element of the retina

Newman, E. A., & Reichenbach, A. (1996) · Trends in Neurosciences · doi:10.1016/0166-2236(96)10040-0 · Cited by 789 · see citing works

Identification of a Natural Soluble Form of the Vascular Endothelial Growth Factor Receptor, FLT-1, and Its Heterodimerization with KDR

Kendall, R. L., Wang, G., & Thomas, K. A. (1996) · Biochemical and Biophysical Research Communications · doi:10.1006/bbrc.1996.1355 · Cited by 730 · see citing works

Retinal angiogenesis in development and disease

Gariano, R. F., & Gardner, T. W. (2005) · Nature · doi:10.1038/nature04482 · Cited by 707 · see citing works

Immune responses to gene therapy vectors: influence on vector function and effector mechanisms

Bessis, N., Garcia-Cozar, F. J., & Boissier, M. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302364 · Cited by 636 · see citing works

Prolonged delivery of brain-derived neurotrophic factor by adenovirus-infected Müller cells temporarily rescues injured retinal ganglion cells

Polo, A. D., Aigner, L., Dunn, R. J., Bray, G. M., & Aguayo, A. J. (1998) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.95.7.3978 · Cited by 430 · see citing works

Development and pathology of the hyaloid, choroidal and retinal vasculature

Saint‐Geniez, M., & D'Amore, P. A. (2004) · The International Journal of Developmental Biology · doi:10.1387/ijdb.041895ms · Cited by 413 · see citing works

Ranibizumab for Treatment of Neovascular Age-Related Macular Degeneration

Heier, J. S., Antoszyk, A. N., PAVAN, P. R., Leff, S. R., Rosenfeld, P. J., Ciulla, T. A., Dreyer, R. F., Gentile, R. C., Sy, J. P., Hantsbarger, G. L., et al. (2006) · Ophthalmology · doi:10.1016/j.ophtha.2005.10.052 · Cited by 360 · see citing works

Blockade of Vascular Endothelial Cell Growth Factor Receptor Signaling Is Sufficient to Completely Prevent Retinal Neovascularization

Ozaki, H., Seo, M., Ozaki, K., Yamada, H., Yamada, E., Okamoto, N., Hofmann, F., Wood, J. M., & Campochiaro, P. A. (2000) · American Journal Of Pathology · doi:10.1016/s0002-9440(10)64773-6 · Cited by 346 · see citing works

Adenoviral Vector-Delivered Pigment Epithelium-Derived Factor for Neovascular Age-Related Macular Degeneration: Results of a Phase I Clinical Trial

Campochiaro, P. A., Nguyen, Q. D., Shah, S. M., Klein, M. L., Holz, E., Frank, R. N., Saperstein, D. A., Gupta, A., Stout, J. T., Macko, J., et al. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.167 · Cited by 343 · see citing works

Oligodeoxynucleotides inhibit retinal neovascularization in a murine model of proliferative retinopathy.

Robinson, G. S., Pierce, E. A., Rook, S. L., Foley, E., Webb, R., & Smith, L. E. H. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.10.4851 · Cited by 337 · 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

Suppression of ocular neovascularization with siRNA targeting VEGF receptor 1

Shen, J., Samul, R., Silva, R. L., Akiyama, H., Liu, H., Saishin, Y., Hackett, S. F., Zinnen, S. P., Kossen, K., Fosnaugh, K. L., et al. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302641 · Cited by 308 · see citing works

Intravitreal Bevacizumab for the Management of Choroidal Neovascularization in Age-related Macular Degeneration

Bashshur, Z. F., Bazarbachi, A. A., Schakal, A., Haddad, Z. A., El-Haibi, C. P., & Noureddin, B. N. (2006) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2006.02.037 · Cited by 305 · see citing works

Pigment epithelium-derived factor suppresses ischemia-induced retinal neovascularization and VEGF-induced migration and growth.

Duh, E. J., Yang, H. S., Suzuma, I., Miyagi, M., Youngman, E. M., Mori, K., Katai, M., Yan, L., Suzuma, K., West, K. A., et al. (2002) · PubMed · Cited by 303 · see citing works

Biology of Adenovirus and Its Use as a Vector for Gene Therapy

McConnell, M. J., & Imperiale, M. J. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1022 · Cited by 230 · see citing works

Inhibitors of Ocular Neovascularization

van Wijngaarden, P., Coster, D. J., & Williams, K. A. (2005) · JAMA · doi:10.1001/jama.293.12.1509 · Cited by 228 · see citing works

Optimization of the helper-dependent adenovirus system for production and potency in vivo

Sandig, V., Youil, R., Bett, A. J., Franlin, L. L., Oshima, M., Maione, D., Wang, F., Metzker, M. L., Savino, R., & Caskey, C. T. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.3.1002 · Cited by 202 · see citing works

Helper-Dependent Adenovirus Vectors Elicit Intact Innate but Attenuated Adaptive Host Immune Responses In Vivo

Muruve, D. A., Cotter, M. J., Zaiss, A. K., White, L. R., Liu, Q., Chan, T., Clark, S. A., Ross, P. J., Meulenbroek, R. A., Mælandsmo, G. M., et al. (2004) · Journal of Virology · doi:10.1128/jvi.78.11.5966-5972.2004 · Cited by 201 · see citing works

Helper-Dependent Adenoviral Vectors for Gene Therapy

Palmer, D. J., & Ng, P. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.1 · Cited by 196 · see citing works

Regression of ocular neovascularization in response to increased expression of pigment epithelium-derived factor.

Mori, K., Gehlbach, P. L., Ando, A., McVey, D., Wei, L. L., & Campochiaro, P. A. (2002) · PubMed · Cited by 195 · see citing works

The range of PaO2 variation determines the severity of oxygen-induced retinopathy in newborn rats.

Penn, J. S., Henry, M. M., Wall, P. T., & Tolman, B. L. (1995) · PubMed · Cited by 166 · see citing works

Dendrimer delivery of an anti-VEGF oligonucleotide into the eye: a long-term study into inhibition of laser-induced CNV, distribution, uptake and toxicity

Marano, R. J., Tóth, I., Wimmer, N., Brankov, M., & Rakoczy, P. E. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302579 · Cited by 164 · see citing works

Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1

Bainbridge, J. W., Mistry, A., de Alwis, M., Paleolog, E. M., Baker, A. H., Thrasher, A. J., & Ali, R. R. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301680 · Cited by 153 · see citing works

Lack of an Immune Response against the Tetracycline-Dependent Transactivator Correlates with Long-Term Doxycycline-Regulated Transgene Expression in Nonhuman Primates after Intramuscular Injection of Recombinant Adeno-Associated Virus

Favre, D., Blouin, V., Provost, N., Spisek, R., Porrot, F., Bohl, D., Marmé, F., Chérel, Y., Salvetti, A., Hurtrel, B., et al. (2002) · Journal of Virology · doi:10.1128/jvi.76.22.11605-11611.2002 · Cited by 150 · see citing works

Gene therapy progress and prospects: the eye

Bainbridge, J. W., Tan, M. H., & Ali, R. R. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302812 · Cited by 144 · see citing works

Age related macular degeneration

Chopdar, A., Chakravarthy, U., & Verma, D. (2003) · BMJ · doi:10.1136/bmj.326.7387.485 · Cited by 139 · 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

Gene therapy progress and prospects: transcription regulatory systems

Toniatti, C., Bujard, H., Cortese, R., & Ciliberto, G. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302251 · Cited by 133 · see citing works

Immune response following intraocular delivery of recombinant viral vectors

Bennett, J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302030 · Cited by 133 · see citing works

Long-term Evaluation of AAV-Mediated sFlt-1 Gene Therapy for Ocular Neovascularization in Mice and Monkeys

Lai, C., Shen, W.-Y., Brankov, M., Lai, Y. K. Y., Barnett, N. L., Lee, S., Yeo, I. Y. S., Mathur, R., Ho, J. E., Pineda, P., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.04.022 · Cited by 124 · 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

Tetracycline-regulated gene expression in the brain.

Mansuy, I., & Bujard, H. (2000) · Current Opinion in Neurobiology · doi:10.1016/s0959-4388(00)00127-6 · Cited by 119 · see citing works

Potential applications for RNAi to probe pathogenesis and develop new treatments for ocular disorders

Campochiaro, P. A. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302653 · Cited by 115 · see citing works

Gene Transfer of a Chimeric Trans-Activator Is Immunogenic and Results in Short-Lived Transgene Expression

Latta-Mahieu, M., Rolland, M., Caillet, C., Wang, M., Kennel, P., Mahfouz, I., Loquet, I., Dedieu, J.-F., Mahfoudi, A., Trannoy, E., et al. (2002) · Human Gene Therapy · doi:10.1089/10430340260201707 · Cited by 114 · see citing works

Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors

Stieger, K., Le Meur, G., Lasne, F., Weber, M., Deschamps, J.-Y., Nivard, D., Mendes-Madeira, A., Provost, N., Martin, L., Moullier, P., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.12.001 · Cited by 110 · see citing works

Ocular neovascularization: a valuable model system

Campochiaro, P. A., & Hackett, S. F. (2003) · Oncogene · doi:10.1038/sj.onc.1206773 · Cited by 100 · see citing works

Wet age-related macular degeneration

KULKARNI, A., & Kuppermann, B. D. (2005) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2005.09.003 · Cited by 93 · see citing works

Immunocytochemical localization of vascular endothelial growth factor in neurons and glial cells of human retina

Famiglietti, E. V., Stopa, E. G., McGookin, E. D., Song, P., Kuo-LeBlanc, V., & Streeten, B. W. (2003) · Brain Research · doi:10.1016/s0006-8993(02)03766-6 · Cited by 91 · 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

Brain-Derived Neurotrophic Factor Gene Delivery to Muller Glia Preserves Structure and Function of Light-Damaged Photoreceptors

Gauthier, R., Joly, S., Pernet, V., Lachapelle, P., & Polo, A. D. (2005) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.05-0362 · Cited by 80 · see citing works

Sustained Phenotypic Correction of Canine Hemophilia B After Systemic Administration of Helper-Dependent Adenoviral Vector

Brunetti‐Pierri, N., Nichols, T. C., McCorquodale, S., Merricks, E. P., Palmer, D. J., Beaudet, A. L., & Ng, P. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.811 · Cited by 79 · see citing works

Novel approaches to ocular drug delivery.

Davis, J. L., Gilger, B. C., & Robinson, M. R. (2004) · PubMed · Cited by 77 · see citing works

Ocular neovascularisation and excessive vascular permeability

Campochiaro, P. A. (2004) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.4.9.1395 · Cited by 77 · see citing works

Tight control of gene expression by a helper-dependent adenovirus vector carrying the rtTA2s-M2 tetracycline transactivator and repressor system

Salucci, V., Scarito, A., Aurisicchio, L., Lamartina, S., Nicolaus, G., Giampaoli, S., Gonzalez-Paz, O., Toniatti, C., Bujard, H., Hillen, W., et al. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301813 · Cited by 76 · see citing works

Construction of an rtTA2s-m2/ttskid-Based transcription regulatory switch that displays no basal activity, good inducibility, and high responsiveness to doxycycline in mice and Non-Human primates

Lamartina, S., Silvi, L., Roscilli, G., Casimiro, D., Simon, A. J., Davies, M., Shiver, J., Rinaudo, C. D., Zampaglione, I., Fattori, E., et al. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(02)00051-5 · Cited by 75 · see citing works

Improved Hepatic Transduction, Reduced Systemic Vector Dissemination, and Long-Term Transgene Expression by Delivering Helper-Dependent Adenoviral Vectors into the Surgically Isolated Liver of Nonhuman Primates

Brunetti‐Pierri, N., Ng, T., Iannitti, D. A., Palmer, D. J., Beaudet, A. L., Finegold, M. J., Carey, K. D., Cioffi, W. G., & Ng, P. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.391 · Cited by 73 · see citing works

Gene Therapy for Ocular Disease

Bennett, J., & Maguire, A. M. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0080 · Cited by 71 · see citing works

Comparative studies on mammalian Muller (retinal glial) cells

Chao, T. I., Grosche, J., Biedermann, B., Francke, M., Pannicke, T., Reichelt, W., WULST, M., Mühle, C., Pritz-Hohmeier, S., Kuhrt, H., et al. (1997) · Journal of Neurocytology · doi:10.1023/a:1018525222826 · Cited by 68 · see citing works

Nonhuman Primate Models for Diabetic Ocular Neovascularization Using AAV2-Mediated Overexpression of Vascular Endothelial Growth Factor

Lebherz, C., Maguire, A. M., Auricchio, A., Tang, W., Alemán, T. S., Wei, Z., Grant, R. L., Cideciyan, A. V., Jacobson, S. G., Wilson, J. M., et al. (2005) · Diabetes · doi:10.2337/diabetes.54.4.1141 · Cited by 68 · see citing works

Long-Term Inducible Gene Expression in the Eye via Adeno-Associated Virus Gene Transfer in Nonhuman Primates

Lebherz, C., Auricchio, A., Maguire, A. M., Rivera, V. M., Tang, W., Grant, R. L., Clackson, T., Bennett, J., & Wilson, J. M. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.178 · Cited by 53 · 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

HLA-A*0201-restricted cytolytic responses to the rtTA transactivator dominant and cryptic epitopes compromise transgene expression induced by the tetracycline on system

Ginhoux, F., Turbant, S., Gross, D. A., Poupiot, J., Marais, T., Lone, Y., Lemonnier, F. A., Firat, H., Perez, N., Danos, O., et al. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.05.012 · Cited by 49 · see citing works

Intraocular adenoviral vector-mediated gene transfer in proliferative retinopathies.

Mori, K., Gehlbach, P. L., Ando, A., Wahlin, K. J., Gunther, V., McVey, D., Wei, L. L., & Campochiaro, P. A. (2002) · PubMed · Cited by 47 · see citing works

Long-term transgene expression in the RPE after gene transfer with a high-capacity adenoviral vector.

Kreppel, F., Luther, T. T., Semkova, I., Schraermeyer, U., & Kochanek, S. (2002) · PubMed · Cited by 46 · see citing works

Canine Adenovirus Vectors for Lung-Directed Gene Transfer: Efficacy, Immune Response, and Duration of Transgene Expression Using Helper-Dependent Vectors

Keriel, A., René, C., Galer, C. E., Zabner, J., & Kremer, E. J. (2006) · Journal of Virology · doi:10.1128/jvi.80.3.1487-1496.2006 · Cited by 42 · see citing works

Inhibition of retinal and choroidal neovascularization by a novel KDR kinase inhibitor.

Kinose, F., Roscilli, G., Lamartina, S., Anderson, K. D., Bonelli, F., Spence, S., Ciliberto, G., Vogt, T., Holder, D., Toniatti, C., et al. (2005) · PubMed · Cited by 42 · see citing works

Gene therapy for proliferative ocular diseases

McFarland, T. J., Zhang, Y., Appukuttan, B., & Stout, J. T. (2004) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.4.7.1053 · Cited by 31 · see citing works

Novel Approaches in the Treatment of Angiogenic Eye Disease

Wegewitz, U. E., Göhring, I., & Spranger, J. (2005) · Current Pharmaceutical Design · doi:10.2174/1381612054367292 · Cited by 20 · see citing works

Current and future pharmacological intervention for diabetic retinopathy

Comer, G. M., & Ciulla, T. A. (2005) · Expert Opinion on Emerging Drugs · doi:10.1517/14728214.10.2.441 · Cited by 19 · see citing works

Gene therapy for ocular neovascularization: a cure in sight

Reich, S. J., & Bennett, J. (2003) · Current Opinion in Genetics & Development · doi:10.1016/s0959-437x(03)00043-1 · Cited by 15 · see citing works

Adeno‐cosmid cloning vectors for regulated gene expression

Giampaoli, S., Nicolaus, G., Delmastro, P., & Cortese, R. (2002) · The Journal of Gene Medicine · doi:10.1002/jgm.286 · Cited by 8 · see citing works

Laser Photocoagulation and, to a Lesser Extent, Photodynamic Therapy Target and Enhance Adenovirus Vector–Mediated Gene Transfer in the Rat Retina

Anzai, K., Yoneya, S., Gehlbach, P. L., Imai, D., Wei, L. L., & Mori, K. (2005) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.05-0593 · Cited by 3 · see citing works

Adenoviral Vector-Delivered Pigment Epithelium-Derived Factor for Neovascular Age-Related Macular Degeneration: Results of a Phase I Clinical Trial

Campochiaro, P. A., Nguyen, Q. D., Shah, S. M., Klein, M. L., Holz, E., Frank, R. N., Saperstein, D. A., Gupta, A., Stout, J. T., Macko, J., et al. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.167 · Cited by 343 · see citing works

Diabetic retinopathy: a complex pathophysiology requiring novel therapeutic strategies

Whitehead, M. A., Wickremasinghe, S., Osborne, A., van Wijngaarden, P., & Martin, K. R. G. (2018) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2018.1545836 · Cited by 198 · see citing works

Vector platforms for gene therapy of inherited retinopathies

Trapani, I., Puppo, A., & Auricchio, A. (2014) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2014.08.001 · Cited by 184 · see citing works

Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection

Hellström, M., Ruitenberg, M. J., Pollett, M. A., Ehlert, E. M. E., Twisk, J., Verhaagen, J., & Harvey, A. R. (2009) · Gene Therapy · doi:10.1038/gt.2008.178 · Cited by 142 · see citing works

Scavenger Receptors and Their Potential as Therapeutic Targets in the Treatment of Cardiovascular Disease

Stephen, S. L., Freestone, K., Dunn, S., Twigg, M., Homer-Vanniasinkam, S., Walker, J. H., Wheatcroft, S., & Ponnambalam, S. (2010) · International Journal of Hypertension · doi:10.4061/2010/646929 · Cited by 120 · see citing works

Treatment of ocular disorders by gene therapy

Solinís, M. Á., del Pozo-Rodríguez, A., Apaolaza, P. S., & Rodríguez-Gascón, A. (2015) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2014.12.022 · Cited by 88 · see citing works

Müller cells as a target for retinal therapy

Devoldere, J., Peynshaert, K., Smedt, S. C. D., & Remaut, K. (2019) · Drug Discovery Today · doi:10.1016/j.drudis.2019.01.023 · Cited by 88 · see citing works

Gene therapy for retinal ganglion cell neuroprotection in glaucoma

Wilson, A. M., & Polo, A. D. (2011) · Gene Therapy · doi:10.1038/gt.2011.142 · Cited by 81 · see citing works

Gene therapy for ocular diseases

Liu, M. M., Tuo, J., & Chan, C.-C. (2011) · British Journal of Ophthalmology · doi:10.1136/bjo.2009.174912 · Cited by 82 · see citing works

A rapid protocol for construction and production of high-capacity adenoviral vectors

Jäger, L., Mück-Häusl, M., Rauschhuber, C., Wolf, N. M., Kay, M. A., & Ehrhardt, A. (2009) · Nature Protocols · doi:10.1038/nprot.2009.4 · Cited by 77 · see citing works

Ocular gene therapy: current progress and future prospects

Colella, P., Cotugno, G., & Auricchio, A. (2008) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2008.11.003 · Cited by 71 · see citing works

Construction and application of adenoviral vectors

Zhang, H., Wang, H., An, Y., & Chen, Z. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.09.004 · Cited by 66 · see citing works

Updates on Gene Therapy for Diabetic Retinopathy

Wang, J.-H., Roberts, G. E., & Liu, G. (2020) · Current Diabetes Reports · doi:10.1007/s11892-020-01308-w · Cited by 62 · see citing works

Retinal transduction profiles by high-capacity viral vectors

Puppo, A., Cesi, G., Marrocco, E., Piccolo, P., Jacca, S., Shayakhmetov, D. M., Parks, R. J., Davidson, B. L., Colloca, S., Brunetti‐Pierri, N., et al. (2014) · Gene Therapy · doi:10.1038/gt.2014.57 · Cited by 58 · see citing works

Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy

Ren, D., Fisson, S., Dalkara, D., & Ail, D. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091973 · Cited by 52 · see citing works

Gene therapy for diabetic retinopathy: Are we ready to make the leap from bench to bedside?

Wang, J.-H., Ling, D. L., Tu, L., van Wijngaarden, P., Dusting, G. J., & Liu, G. (2017) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2017.01.003 · Cited by 51 · see citing works

Study of the Efficacy, Biodistribution, and Safety Profile of Therapeutic Gutless Adenovirus Vectors as a Prelude to a Phase I Clinical Trial for Glioblastoma

Muhammad, A. K. M. G., Puntel, M., Candolfi, M., Salem, A., Yagiz, K., Farrokhi, C. B., Kroeger, K. M., Xiong, W., Curtin, J. F., Liu, C., et al. (2010) · Clinical Pharmacology & Therapeutics · doi:10.1038/clpt.2009.260 · Cited by 46 · see citing works

Gene delivery to the retina: focus on non-viral approaches

Naik, R. J., Mukhopadhyay, A., & Ganguli, M. (2008) · Drug Discovery Today · doi:10.1016/j.drudis.2008.09.012 · Cited by 45 · see citing works

Barriers for retinal gene therapy: separating fact from fiction.

Kumar-Singh, R. (2008) · Vision Research · doi:10.1016/j.visres.2008.05.005 · Cited by 43 · see citing works

Gene therapy and transplantation in the retinofugal pathway

Harvey, A. R., Hellström, M., & Rodger, J. (2009) · Progress in brain research · doi:10.1016/s0079-6123(09)17510-6 · Cited by 42 · see citing works

Gene Therapy of ABCA4-Associated DiseasesPreprint

Auricchio, A., Trapani, I., & Allikmets, R. L. (2015) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a017301 · Cited by 41 · see citing works

Novel stem cell and gene therapy in diabetic retinopathy, age related macular degeneration, and retinitis pigmentosa

Ludwig, P. E., Freeman, S. C., & Janot, A. C. (2019) · International Journal of Retina and Vitreous · doi:10.1186/s40942-019-0158-y · Cited by 41 · see citing works

Evaluation of adenovirus-delivered human CD59 as a potential therapy for AMD in a model of human membrane attack complex formation on murine RPE

Ramo, K., Cashman, S. M., & Kumar-Singh, R. (2008) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.08-2025 · Cited by 38 · see citing works

Current treatment strategies and nanocarrier based approaches for the treatment and management of diabetic retinopathy

Selvaraj, K., Kuppusamy, G., Karri, V. V. S. R., Barauah, U. K., Ravisankar, V., & Jojo, G. M. (2017) · Journal of drug targeting · doi:10.1080/1061186x.2017.1280809 · Cited by 37 · see citing works

Gene Therapy for Inherited Retinal Degeneration

Arbabi, A., Liu, A., & Ameri, H. (2019) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2018.0087 · Cited by 30 · see citing works

Safety profile, efficacy, and biodistribution of a bicistronic high-capacity adenovirus vector encoding a combined immunostimulation and cytotoxic gene therapy as a prelude to a phase I clinical trial for glioblastoma

Puntel, M., Ghulam Muhammad, A. K. M., Farrokhi, C. B., VanderVeen, N. T., Paran, C. W., Appelhans, A., Kroeger, K. M., Salem, A., Lacayo, L., Pechnick, R. N., et al. (2013) · Toxicology and Applied Pharmacology · doi:10.1016/j.taap.2013.02.001 · Cited by 28 · see citing works

Pathophysiology and management of diabetic retinopathy

El‐Asrar, A. M. A., Al‐Mezaine, H. S., & Ola, M. S. (2009) · Expert Review of Ophthalmology · doi:10.1586/eop.09.52 · Cited by 26 · see citing works

Safety Profile of Gutless Adenovirus Vectors Delivered into the Normal Brain Parenchyma: Implications for a Glioma Phase 1 Clinical Trial

Muhammad, A. K. M. G., Xiong, W., Puntel, M., Farrokhi, C. B., Kroeger, K. M., Salem, A., Lacayo, L., Pechnick, R. N., Kelson, K. R., Palmer, D. J., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.060 · Cited by 24 · see citing works

Decay Accelerating Factor (CD55)–Mediated Attenuation of Complement: Therapeutic Implications for Age-Related Macular Degeneration

Kelly, N., Cashman, S. M., Sweigard, J. H., & Kumar‐Singh, R. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-5887 · Cited by 19 · see citing works

The Role of Pharmacogenetics and Advances in Gene Therapy in the Treatment of Diabetic Retinopathy

Agarwal, A. K., Ingham, S. A., Harkins, K. A., Diana, V., & Nguyen, Q. D. (2016) · Pharmacogenomics · doi:10.2217/pgs.15.173 · Cited by 18 · see citing works

Experiment-Based Interventions to Diabetic Retinopathy: Present and Advances

Liu, S., Ju, Y., & Gu, P. D. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23137005 · Cited by 16 · see citing works

Unconventional avenues to decelerate diabetic retinopathy

Takkar, B., Sheemar, A., Jayasudha, R., Soni, D., Narayanan, R., Venkatesh, P., Shivaji, S., & Das, T. (2022) · Survey of Ophthalmology · doi:10.1016/j.survophthal.2022.06.004 · Cited by 16 · 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

Diabetic retinopathy in focus: Update on treatment advances, pharmaceutical approaches, and new technologies

Ciorba, A. L., Saber, S., Abdelhamid, A. M., Keshk, N., Elnaghy, F., Elmorsy, E. A., Abu‐Khudir, R., Hamad, R. S., Abdel‐Reheim, M. A., Farrag, A. A. M., et al. (2025) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2025.107307 · Cited by 14 · see citing works

Subretinal Gene Delivery Using Helper-Dependent Adenoviral Vectors

Wu, L., Lam, S., Cao, H., Guan, R., Duan, R., van der Kooy, D., Bremner, R., Molday, R. S., & Hu, J. (2011) · Cell & Bioscience · doi:10.1186/2045-3701-1-15 · Cited by 13 · see citing works

Neovascularization: Ocular Diseases, Animal Models and Therapies

Cai, X., Sezate, S. A., & McGinnis, J. F. (2011) · Advances in experimental medicine and biology · doi:10.1007/978-1-4614-0631-0_32 · Cited by 13 · see citing works

Helper-dependent adenoviral vectors

Brunetti‐Pierri, N., & Ng, P. (2010) · Springer eBooks · doi:10.1007/978-3-0346-0165-8_13 · Cited by 13 · see citing works

Adenovirus-mediated delivery of CD46 attenuates the alternative complement pathway on RPE: implications for age-related macular degeneration

Sweigard, J. H., Cashman, S. M., & Kumar‐Singh, R. (2011) · Gene Therapy · doi:10.1038/gt.2011.6 · Cited by 12 · see citing works

Helper-Dependent Adenoviral Vectors

Brunetti‐Pierri, N., & Ng, P. (2016) · Elsevier eBooks · doi:10.1016/b978-0-12-800276-6.00017-6 · Cited by 8 · see citing works

Gene Therapies for Neovascular Age-Related Macular DegenerationPreprint

Pechan, P. A., Wadsworth, S. C., & Scaria, A. (2014) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a017335 · Cited by 7 · see citing works

The Effects of Nicotinamide Adenine Dinucleotide Phosphate (NADPH) Oxidase and Erythropoietin, and Their Interactions in Angiogenesis: Implications in Retinopathy of Prematurity

Cung, T., Wang, H., & Hartnett, M. E. (2022) · Cells · doi:10.3390/cells11121951 · Cited by 5 · see citing works

Gene Therapy for Retinal Diseases

Gopinath, C., Sarkar, S., & Ghosh, A. (2021) · Essentials in ophthalmology/Essentials ophthalmology · doi:10.1007/978-981-15-9184-6_30 · Cited by 3 · see citing works

Gene Therapy for Diabetic Retinopathy in Animal Models and Humans

Mizukami, H. (2011) · The Open Diabetes Journal · doi:10.2174/1876524601104010119 · Cited by 3 · see citing works

Electroporation in the Rodent Retina In Vivo and In Vitro

Matsuda, T. (2015) · Neuromethods · doi:10.1007/978-1-4939-2459-2_4 · Cited by 3 · see citing works

Gene Therapy in Diabetic Retinopathy

Raman, R., Swathipriya, N., & Sripriya, S. (2022) · Springer eBooks · doi:10.1007/978-981-16-4247-0_15 · Cited by 3 · see citing works

[Gene therapy for hereditary ophthalmological diseases: Advances and future perspectives].

Chacon-Camacho, O. F., Astorga-Carballo, A., & Zenteno, J. C. (2015) · PubMed · Cited by 2 · see citing works

Helper-Dependent Adenoviral Vectors for Gene Therapy

Brunetti‐Pierri, N., & Ng, P. (2015) · CRC Press eBooks · doi:10.1201/b18002-5 · Cited by 2 · see citing works

Clinical Application of Drug Delivery Systems for Treating AMD

Kuno, N., & Fujii, S. (2012) · InTech eBooks · doi:10.5772/31861 · Cited by 1 · see citing works

Gene therapy for Stargardt and other ABCA4-related diseases: lessons from RPE65-LCA trials

Silva, N. F. A. (2010) · Portuguese National Funding Agency for Science, Research and Technology (RCAAP Project by FCT) · Cited by 1 · see citing works

Recent advances on modeling retinal disease: Towards efficient gene/drug therapy

Dolatabadi, E. N., Zaky, M. R. A., Abbas, F. H., Milani, A. E., André, H., & Alizadeh, E. (2025) · Experimental Eye Research · doi:10.1016/j.exer.2025.110416 · Cited by 1 · see citing works

Combinations of gene therapy, tissue transplantation and pharmacotherapeutic techniques in visual system repair

Hellström, M. (2010) · UWA Profiles and Research Repository (UWA) · Cited by 1 · see citing works

Viral vector-mediated RNA interference in the retina

Georgiadis, A. (2010) · UCL Discovery (University College London)

EUROPEAN SCHOOL OF MOLECULAR MEDICINE

Napoli, S. D. (2010)

Anti-Angiogenic Gene Therapy: Basic Science and Challenges for Translation into the Clinic

Lange, C., & Bainbridge, J. W. (2016) · Essentials in ophthalmology/Essentials ophthalmology · doi:10.1007/978-3-319-24097-8_11

Development of Helper-Dependent Adenoviral Vectors for Gene Therapy for Inherited Retinal Diseases

Lam, S. (2015) · TSpace (University of Toronto)

Safety Profile of Gutless Adenovirus Vectors Delivered into the Normal Brain Parenchyma: Implications for a Glioma Phase I Clinical Trial

Castro, M. G., Xiong, W., Puntel, M., Farrokhi, C. B., Kroeger, K. M., Pechnick, R. N., Ng, P., Lowenstein, P. R., Muhammad, A. G., Salem, A., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hum.2012.060

Pathophysiology and gene therapy of the optic neuropathy in Wolfram Syndrome

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

Helper-Dependent Adenoviral Vectors: Recent Advances and Emerging Applications

Alshawabkeh, M., Ehrke‐Schulz, E., Zhang, W., & Ehrhardt, A. (2026) · Human Gene Therapy · doi:10.1177/10430342261460742

Ocular gene therapy targeting retinal angiogenesis and vascular leakage: translational and clinical evidence from a systematic review and meta-analysis

Chen, K.-Y., Chan, H.-C., & Chan, C.-M. (2026) · Angiogenesis · doi:10.1007/s10456-026-10050-y

Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1

Bainbridge, J. W., Mistry, A., de Alwis, M., Paleolog, E. M., Baker, A. H., Thrasher, A. J., & Ali, R. R. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301680 · Cited by 153 · see citing works

Gene therapy for ocular angiogenesis

Bainbridge, J. W., Mistry, A., Thrasher, A. J., & Ali, R. R. (2003) · UCL Discovery (University College London)

Adeno‐associated virus mediated gene therapy for vascular retinopathies

Raisler, B. J., Deng, W., Berns, K. I., & Hauswirth, W. W. (2005) · Laboratory techniques in biochemistry and molecular biology · doi:10.1016/s0075-7535(05)31005-9 · Cited by 1 · 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

Subretinal Gene Delivery Using Helper-Dependent Adenoviral Vectors

Wu, L., Lam, S., Cao, H., Guan, R., Duan, R., van der Kooy, D., Bremner, R., Molday, R. S., & Hu, J. (2011) · Cell & Bioscience · doi:10.1186/2045-3701-1-15 · Cited by 13 · see citing works