Ocular Inflammation and Treatment Emergent Adverse Events in Retinal Gene Therapy

Mehta, Neesurg; Robbins, Deborah A.; Yiu, Glenn

doi:10.1097/iio.0000000000000366

Cited by 33Retinal Development and DisordersOcular Diseases and Behçet’s SyndromeOcular Infections and Treatments

Ocular Inflammation and Treatment Emergent Adverse Events in Retinal Gene Therapy

Open access

Mehta, Neesurg, Robbins, Deborah A., Yiu, Glenn·2021·International Ophthalmology Clinics

DOI 10.1097/iio.0000000000000366PMC PMC8259781

Open this paper in CoChat — free account

Abstract

International Ophthalmology Clinics: Summer 2021 - Volume 61 - Issue 3 - p 151-177 doi: 10.1097/IIO.0000000000000366

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Integrity signals

Registry

Crossref · 10.1097/iio.0000000000000366

Cites

123 of 123 references verified0 citation errors · 0 not checkable

Statistics

Claims the paper makes

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Rituximab could be administered around the time of first-eye treatment to prevent the resulting increase in neutralising antibody titres by temporarily depleting B cell populations.
“For example, rituximab, an anti-CD20 monoclonal anti body, could be administered around the time of first eye treatment to prevent the resulting increase in NAb titres by temporarily depleting B cell populations (Mehta et al., 2021).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Mehta et al., 2021
The Mitigation of Humoral Immune Responses section says that targeting B cells can limit humoral responses and that anti-CD20 treatment was partially effective in reducing AAV neutralising antibodies in some rheumatoid arthritis patients. The Route of Administration & Biodistribution section reports increased antibody titres after first-eye treatment in dogs and nonhuman primates. The paper does not name rituximab or propose giving it around first-eye treatment to prevent that increase; page numbers are unavailable in our copy. (LLM, conf=0.86)
Experimental — accuracy not yet measured
Dose-dependent immune responses were true in both subretinal and intravitreal deliveries, although the same viral dose induced lower immune responses when delivered via the subretinal route.
“This was true in both subretinal and intravitreal deliveries, although the same viral dose induced lower immune responses when delivered via the subretinal route.91,105–107”
From Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses (2022) · cites this paper as [107]
Pre-clinical and clinical studies have indicated that gene therapy with AAV can induce dose-dependent innate and adaptive immune responses.
“Pre-clinical and clinical studies have indicated that gene therapy with AAV can induce dose-dependent innate and adaptive immune responses.”
From Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses (2022) · cites this paper as [107]

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

The self-check needs the paper’s full text.

1 retracted

RETRACTED: Activation of the NF-κB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy
Jayandharan, G. R., Aslanidi, G., Martino, A. T., Jahn, S. C., Perrin, G. Q., Herzog, R. W., & Srivastava, A. (2011) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1012753108 · Cited by 72 · see citing works
Retracted
Toll-Like Receptor Signaling Pathways
Kawasaki, T., & Kawai, T. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00461 · Cited by 3,250 · 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
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163 · see citing works
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
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
Raper, S. E., Chirmule, N. B., Lee, F. S., Wivel, N. A., Bagg, A., Gao, G., Wilson, J. M., & Batshaw, M. L. (2003) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2003.08.016 · Cited by 1,502 · see citing works
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors
Boutin, S., Monteilhet, V., Veron, P., Leborgne, C., Benveniste, O., Montus, M. F., & Masurier, C. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.182 · Cited by 970 · see citing works
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
Mingozzi, F., & High, K. A. (2013) · Blood · doi:10.1182/blood-2013-01-306647 · Cited by 955 · see citing works
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses
Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J., & Wilson, J. M. (2009) · The Journal of Infectious Diseases · doi:10.1086/595830 · Cited by 808 · see citing works
Immune Responses to Viral Gene Therapy Vectors
Shirley, J. L., de Jong, Y. P., Terhorst, C., & Herzog, R. W. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.01.001 · Cited by 788 · see citing works
Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans
Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine · doi:10.1038/nm1549 · Cited by 748 · 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
Dystrophin Immunity in Duchenne's Muscular Dystrophy
Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000228 · Cited by 655 · 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
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy
Maguire, A. M., Russell, S., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., Marshall, K. A., et al. (2019) · Ophthalmology · doi:10.1016/j.ophtha.2019.06.017 · 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
Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors
Zaiss, A.-K., Liu, Q., Bowen, G. P., Wong, N. C. W., Bartlett, J. S., & Muruve, D. A. (2002) · Journal of Virology · doi:10.1128/jvi.76.9.4580-4590.2002 · Cited by 394 · see citing works
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Mingozzi, F., Anguela, X. M., Pavani, G., Chen, Y., Davidson, R. J., Hui, D. J., Yazicioglu, M., Elkouby, L., Hinderer, C. J., Faella, A., et al. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005795 · Cited by 355 · see citing works
Cd1-Reactive Natural Killer T Cells Are Required for Development of Systemic Tolerance through an Immune-Privileged Site
Sonoda, K., Exley, M. A., Snapper, S. B., Balk, S. P., & Stein‐Streilein, J. (1999) · The Journal of Experimental Medicine · doi:10.1084/jem.190.9.1215 · Cited by 351 · see citing works
Adenovirus: The First Effective In Vivo Gene Delivery Vector
Crystal, R. G. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.2527 · Cited by 343 · see citing works
The <scp>AIM</scp>2 inflammasome: Sensor of pathogens and cellular perturbations
Lugrin, J., & Martinon, F. (2018) · Immunological Reviews · doi:10.1111/imr.12618 · Cited by 333 · see citing works
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
Zhu, J., Huang, X., & Yang, Y. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37607 · Cited by 330 · see citing works
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
Jiang, H., Couto, L. B., Patarroyo-White, S., Liu, T., Nagy, D., Vargas, J. A., Zhou, S., Scallan, C. D., Sommer, J., Vijay, S., et al. (2006) · Blood · doi:10.1182/blood-2006-04-017913 · Cited by 330 · see citing works
Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in Vivo Cell Tracking Experiments
Ansari, A. M., Ahmed, A. K., Matsangos, A. E., Lay, F., Born, L. J., Marti, G. P., Harmon, J. W., & Sun, Z. (2016) · Stem Cell Reviews and Reports · doi:10.1007/s12015-016-9670-8 · Cited by 324 · see citing works
Adeno-associated viruses undergo substantial evolution in primates during natural infections
Gao, G., Alvira, M. R., Somanathan, S., Lü, Y., Vandenberghe, L. H., Rux, J. J., Calcedo, R., Sanmiguel, J. C., Abbas, Z., & Wilson, J. M. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0937739100 · Cited by 323 · see citing works
AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
Mingozzi, F., Meulenberg, J. J., Hui, D. J., Basner-Tschakarjan, E., Hasbrouck, N. C., Edmonson, S. A., Hutnick, N. A., Betts, M. R., Kastelein, J. J., Stroes, E. S., et al. (2009) · Blood · doi:10.1182/blood-2008-07-167510 · Cited by 295 · see citing works
Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice
Scallan, C. D., Jiang, H., Liu, T., Patarroyo-White, S., Sommer, J. M., Zhou, S., Couto, L. B., & Pierce, G. F. (2006) · Blood · doi:10.1182/blood-2005-08-3229 · Cited by 271 · see citing works
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery
Cukras, C., Wiley, H. E., Jeffrey, B. G., Sen, H. N., Turriff, A., Zeng, Y., Vijayasarathy, C., Marangoni, D., Ziccardi, L., Kjellstrom, S., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.05.025 · Cited by 268 · 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
CpG-depleted adeno-associated virus vectors evade immune detection
Faust, S. M., Bell, P., Cutler, B. J., Ashley, S. N., Zhu, Y., Rabinowitz, J. E., & Wilson, J. M. (2013) · Journal of Clinical Investigation · doi:10.1172/jci68205 · Cited by 254 · see citing works
Cutting Edge: Role of Macrophage Migration Inhibitory Factor in Inhibiting NK Cell Activity and Preserving Immune Privilege
Apte, R. S., Sinha, D., Mayhew, E. S., Wistow, G. J., & Niederkorn, J. Y. (1998) · The Journal of Immunology · doi:10.4049/jimmunol.160.12.5693 · Cited by 253 · see citing works
Studies on the induction of anterior chamber‐associated immune deviation (ACAID) III. Induction of ACAID depends upon intraocular transforming growth factor‐β
Wilbanks, G. A., Mammolenti, M., & Streilein, J. W. (1992) · European Journal of Immunology · doi:10.1002/eji.1830220125 · Cited by 249 · see citing works
AAV cis -regulatory sequences are correlated with ocular toxicity
Xiong, W., Wu, D. M., Xue, Y., Wang, S. K., Chung, M. J., Ji, X., Rana, P., Zhao, S. R., Mai, S., & Cepko, C. L. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1821000116 · Cited by 249 · see citing works
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver
Martino, A. T., Suzuki, M., Markusic, D. M., Zolotukhin, I., Ryals, R. C., Moghimi, B., Ertl, H. C. J., Muruve, D. A., Lee, B., & Herzog, R. W. (2011) · Blood · doi:10.1182/blood-2010-10-314518 · Cited by 246 · see citing works
Humoral immune response to AAV
Calcedo, R., & Wilson, J. M. (2013) · Frontiers in Immunology · doi:10.3389/fimmu.2013.00341 · Cited by 243 · 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
Immune responses to retinal gene therapy using adeno-associated viral vectors – implications for treatment success and safety
Bucher, K., Rodríguez-Bocanegra, E., Dauletbekov, D., & Fischer, M. D. (2021) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2020.100915 · Cited by 242 · see citing works
Unraveling the complex story of immune responses to AAV vectors trial after trial.
Vandamme, C., Adjali, O., & Mingozzi, F. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.150 · Cited by 234 · see citing works
Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial
Heier, J. S., Kherani, S., Desai, S., Dugel, P., Kaushal, S., Cheng, S. H., Delacono, C., Purvis, A., Richards, S., Le-Halpere, A., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)30979-0 · Cited by 229 · see citing works
Identification of alpha-melanocyte stimulating hormone as a potential immunosuppressive factor in aqueous humor
Taylor, A. W., Streilein, J. W., & Cousins, S. W. (1992) · Current Eye Research · doi:10.3109/02713689208999545 · Cited by 226 · see citing works
Toll–Like Receptor 2–Mediated Innate Immune Response in Human Nonparenchymal Liver Cells Toward Adeno–Associated Viral Vectors
Hösel, M., Broxtermann, M., Janicki, H., Esser, K., Arzberger, S., Hartmann, P., Gillen, S., Kleeff, J., Stabenow, D., Odenthal, M., et al. (2012) · Hepatology · doi:10.1002/hep.24625 · Cited by 219 · see citing works
Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors
Wang, L., Calcedo, R., Bell, P., Lin, J., Grant, R. L., Siegel, D. L., & Wilson, J. M. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.031 · Cited by 212 · see citing works
A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8
Monteilhet, V., Saheb, S., Boutin, S., Leborgne, C., Veron, P., Montus, M.-F., Moullier, P., Benveniste, O., & Masurier, C. (2011) · Molecular Therapy · doi:10.1038/mt.2011.108 · Cited by 206 · see citing works
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Fitzpatrick, Z., Leborgne, C., Barbon, E., Masat, E., Ronzitti, G., van Wittenberghe, L., Vignaud, A., Collaud, F., Charles, S., Simon Sola, M., et al. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.02.003 · Cited by 192 · see citing works
Efficacy and Safety of Suprachoroidal CLS-TA for Macular Edema Secondary to Noninfectious Uveitis
Yeh, S., Khurana, R. N., Shah, M., Henry, C. R., Wang, R. C., Kissner, J. M., Ciulla, T. A., & Noronha, G. (2020) · Ophthalmology · doi:10.1016/j.ophtha.2020.01.006 · 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
Differential expression of the complement regulatory proteins in the human eye.
Bora, N. S., Gobleman, C. L., Atkinson, J. P., Pepose, J. S., & Kaplan, H. J. (1993) · PubMed · Cited by 186 · see citing works
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia
Xue, K., Jolly, J. K., Barnard, A. R., Rudenko, A., Salvetti, A. P., Patrício, M. I., Edwards, T. L., Groppe, M., Orlans, H. O., Tolmachova, T., et al. (2018) · Nature Medicine · doi:10.1038/s41591-018-0185-5 · Cited by 185 · see citing works
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential.
(2008) · PubMed Central · Cited by 183 · see citing works
Subretinal space and vitreous cavity as immunologically privileged sites for retinal allografts.
Jiang, L. Q., Jorquera, M., & Streilein, J. W. (1993) · PubMed · Cited by 182 · see citing works
Efficacy and Safety of rAAV2-ND4 Treatment for Leber’s Hereditary Optic Neuropathy
Wan, X., Pei, H., Zhao, M.-J., Yang, S., Hu, W.-K., He, H., Ma, S.-Q., Zhang, G., Dong, X.-Y., Chen, C., et al. (2016) · Scientific Reports · doi:10.1038/srep21587 · Cited by 182 · see citing works
Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors
Kruzik, A., Fetahagic, D., Hartlieb, B., Dorn, S., Koppensteiner, H., Horling, F. M., Scheiflinger, F., Reipert, B. M., & de la Rosa, M. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.05.014 · Cited by 182 · see citing works
Innate Immune Responses to AAV Vectors
Rogers, G. L., Martino, A. T., Aslanidi, G. V., Jayandharan, G. R., Srivastava, A., & Herzog, R. W. (2011) · Frontiers in Microbiology · doi:10.3389/fmicb.2011.00194 · Cited by 178 · see citing works
Studies on the induction of anterior chamber-associated immune deviation (ACAID). 1. Evidence that an antigen-specific, ACAID-inducing, cell-associated signal exists in the peripheral blood
Wilbanks, G. A., & Streilein, J. W. (1991) · The Journal of Immunology · doi:10.4049/jimmunol.146.8.2610 · Cited by 177 · see citing works
Complement Is an Essential Component of the Immune Response to Adeno-Associated Virus Vectors
Zaiss, A. K., Cotter, M. J., White, L. R., Clark, S. A., Wong, N. C. W., Holers, V. M., Bartlett, J. S., & Muruve, D. A. (2008) · Journal of Virology · doi:10.1128/jvi.01990-07 · Cited by 177 · see citing works
Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial
Fischer, M. D., Michalakis, S., Wilhelm, B., Zobor, D., Muehlfriedel, R., Kohl, S., Weisschuh, N., Ochakovski, G. A., Klein, R., Schoen, C., et al. (2020) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2020.1032 · Cited by 177 · see citing works
Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience
Dimopoulos, I. S., Hoang, S. C., Radziwon, A., Binczyk, N. M., Seabra, M. C., MacLaren, R. E., Somani, R., Tennant, M. T. S., & MacDonald, I. M. (2018) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2018.06.011 · Cited by 175 · see citing works
Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery
Nidetz, N. F., McGee, M. C., Tse, L. V., Li, C., Cong, L., Li, Y., & Huang, W. (2020) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2019.107453 · Cited by 174 · see citing works
Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates
Kotterman, M. A., Yin, L., Strazzeri, J. M., Flannery, J. G., Merigan, W. H., & Schaffer, D. V. (2015) · Gene Therapy · doi:10.1038/gt.2014.115 · Cited by 169 · 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
Choroideremia gene therapy phase 2 clinical trial: 24-month results
Lam, B. L., Davis, J. L., Gregori, N. Z., MacLaren, R. E., Girach, A., Verriotto, J. D., Rodriguez, B., Rosa, P. R., Zhang, X., & Feuer, W. J. (2019) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2018.09.012 · Cited by 161 · see citing works
Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates
Yiu, G., Chung, S. H., Mollhoff, I. N., Nguyen, U. T., Thomasy, S. M., Yoo, J., Taraborelli, D., & Noronha, G. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.01.002 · Cited by 153 · see citing works
Self-citation
Phase 2a randomized clinical trial: safety and post hoc analysis of subretinal rAAV.sFLT-1 for wet age-related macular degeneration
Constable, I. J., Pierce, C. M., Lai, C.-M., Magno, A. L., Degli-Esposti, M. A., French, M. A., McAllister, I. L., Butler, S., Barone, S. B., Schwartz, S. D., et al. (2016) · EBioMedicine · doi:10.1016/j.ebiom.2016.11.016 · Cited by 152 · see citing works
Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina
Ramachandran, P. S., Lee, V., Wei, Z., Song, J. Y., Casal, G., Cronin, T., Willett, K., Huckfeldt, R., Morgan, J. I. W., Aleman, T. S., et al. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.111 · Cited by 147 · see citing works
AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye
Reichel, F. F., Dauletbekov, D. L., Klein, R., Peters, T., Ochakovski, G. A., Seitz, I. P., Wilhelm, B., Ueffing, M., Biel, M., Wissinger, B., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.08.018 · Cited by 145 · see citing works
AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression
Ding, K., Shen, J., Hafiz, Z., Hackett, S. F., Silva, R. L. E., Khan, M., Lorenc, V. E., Chen, D., Chadha, R., Zhang, M., et al. (2019) · Journal of Clinical Investigation · doi:10.1172/jci129085 · Cited by 141 · see citing works
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness
Amado, D., Mingozzi, F., Hui, D., Bennicelli, J. L., Wei, Z., Chen, Y., Bote, E., Grant, R. L., Golden, J. A., Narfstrom, K., et al. (2010) · Science Translational Medicine · doi:10.1126/scitranslmed.3000659 · Cited by 140 · see citing works
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
Mingozzi, F., Chen, Y., Edmonson, S. C., Zhou, S., Thurlings, R. M., Tak, P. P., High, K. A., & Vervoordeldonk, M. J. (2013) · Gene Therapy · doi:10.1038/gt.2012.55 · Cited by 138 · see citing works
Safety and long-term efficacy of AAV4 gene therapy in patients with RPE65 leber congenital amaurosis
Le Meur, G., Lebranchu, P., Billaud, F., Adjali, O., Schmitt, S., Bézieau, S., Péréon, Y., Valabregue, R., Ivan, C., Darmon, C., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.014 · Cited by 137 · see citing works
Evidence that retinal pigment epithelium functions as an immune-privileged tissue.
Wenkel, H., & Streilein, J. W. (2000) · PubMed · Cited by 134 · see citing works
Analysis of immune deviation elicited by antigens injected into the subretinal space.
Wenkel, H., & Streilein, J. W. (1998) · PubMed · Cited by 132 · see citing works
Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells
Rogers, G. L., Shirley, J. L., Zolotukhin, I., Kumar, S. R. P., Sherman, A., Perrin, G. Q., Hoffman, B. E., Srivastava, A., Basner-Tschakarjan, E., Wallet, M. A., et al. (2017) · Blood · doi:10.1182/blood-2016-11-751040 · Cited by 131 · see citing works
Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A
Dyka, F. M., Boye, S. L., Chiodo, V. A., Hauswirth, W. W., & Boye, S. E. (2014) · Human Gene Therapy Methods · doi:10.1089/hgtb.2013.212 · Cited by 128 · see citing works
Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction
Shao, W., Earley, L. F., Chai, Z., Chen, X., Sun, J., He, T., Deng, M., Hirsch, M. L., Ting, J., Samulski, R. J., et al. (2018) · JCI Insight · doi:10.1172/jci.insight.120474 · Cited by 119 · see citing works
Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction
Finn, J. D., Hui, D., Downey, H. D., Dunn, D., Pien, G. C., Mingozzi, F., Zhou, S., & High, K. A. (2010) · Molecular Therapy · doi:10.1038/mt.2009.257 · Cited by 115 · 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
Characterization of the Choroid-Scleral Junction and Suprachoroidal Layer in Healthy Individuals on Enhanced-Depth Imaging Optical Coherence Tomography
Yiu, G. C., Pecen, P. E., Sarin, N., Chiu, S. J., Farsiu, S., Mruthyunjaya, P., & Toth, C. A. (2013) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2013.7288 · Cited by 113 · see citing works
Self-citation
Superior retinal gene transfer and biodistribution profile of subretinal versus intravitreal delivery of AAV8 in nonhuman primates
Seitz, I. P., Michalakis, S., Wilhelm, B., Reichel, F. F., Ochakovski, G. A., Zrenner, E., Ueffing, M., Biel, M., Wissinger, B., Bartz-Schmidt, K. U., et al. (2017) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.17-22473 · Cited by 108 · see citing works
Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the ND4 Gene
Bouquet, C., Vignal Clermont, C., Galy, A., Fitoussi, S., Blouin, L., Munk, M. R., Valero, S., Meunier, S., Katz, B., Sahel, J. A., et al. (2019) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2018.6902 · Cited by 102 · see citing works
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice
Cantore, A., Nair, N., Della Valle, P., Di Matteo, M., Màtrai, J., Sanvito, F., Brombin, C., Di Serio, C., D'Angelo, A., Chuah, M., et al. (2012) · Blood · doi:10.1182/blood-2012-05-432591 · Cited by 101 · see citing works
Immunosuppressive Properties of the Pigmented Epithelial Cells and the Subretinal Space
Zamiri, P., Sugita, S., & Streilein, J. W. (2007) · KARGER eBooks · doi:10.1159/000099259 · Cited by 100 · see citing works
Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa
Conlon, T. J., Deng, W., Erger, K. E., Cossette, T. L., Pang, J., Ryals, R. C., Clément, N., Cleaver, B. D., McDoom, I., Boye, S. L., et al. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.037 · Cited by 99 · see citing works
Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8+ T Cell Response to Vector Encoded Proteins
Mays, L. E., Vandenberghe, L. H., Xiao, R., Bell, P. L., Nam, H.-J., Agbandje‐McKenna, M., & Wilson, J. M. (2009) · The Journal of Immunology · doi:10.4049/jimmunol.0803965 · Cited by 98 · see citing works
Immune Response Mechanisms against AAV Vectors in Animal Models
Martino, A. T., & Markusic, D. M. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.12.008 · Cited by 97 · see citing works
rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters
Beltran, W. A., Boye, S. L., Boye, S. E., Chiodo, V. A., Lewin, A. S., Hauswirth, W. W., & Aguirre, G. D. (2010) · Gene Therapy · doi:10.1038/gt.2010.56 · Cited by 96 · see citing works
Dosage thresholds and influence of transgene cassette in adeno-associated virus–related toxicity
Khabou, H., Cordeau, C., Pacot, L., Fisson, S., & Dalkara, D. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.144 · Cited by 93 · see citing works
Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia: A Randomized Clinical Trial
Fischer, M. D., Ochakovski, G. A., Beier, B., Seitz, I. P., Vaheb, Y., Kortuem, C., Reichel, F. F. L., Kuehlewein, L., Kahle, N. A., Peters, T., et al. (2019) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2019.3278 · Cited by 92 · see citing works
Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays
Buck, T., & Wijnholds, J. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21124197 · Cited by 92 · see citing works
Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo
Gabriel, N., Hareendran, S., Sen, D., Gadkari, R. A., Sudha, G., Selot, R., Hussain, M., Dhaksnamoorthy, R., Samuel, R., Srinivasan, N., et al. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.194 · Cited by 90 · see citing works
Striatal Delivery of rAAV-hAADC to Rats with Preexisting Immunity to AAV
Sanftner, L. M., Suzuki, B. M., Doroudchi, M. M., Feng, L., McClelland, A., Forsayeth, J. R., & Cunningham, J. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2003.12.005 · Cited by 89 · see citing works
The analysis of systemic tolerance elicited by antigen inoculation into the vitreous cavity: vitreous cavity‐associated immune deviation
Sonoda, K., Sakamoto, T., Qiao, H., Hisatomi, T., Oshima, T., Tsutsumi‐Miyahara, C., Exley, M., Balk, S. P., Taniguchi, M., & Ishibashi, T. (2005) · Immunology · doi:10.1111/j.1365-2567.2005.02239.x · Cited by 87 · see citing works
Humoral immune response after intravitreal but not after subretinal AAV8 in primates and patients
Reichel, F. F., Peters, T., Wilhelm, B., Biel, M., Ueffing, M., Wissinger, B., Bartz-Schmidt, K. U., Klein, R., Michalakis, S., Fischer, M. D., et al. (2018) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.17-22494 · Cited by 86 · see citing works
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
Hui, D. J., Edmonson, S. C., Podsakoff, G. M., Pien, G. C., Ivanciu, L., Camire, R. M., Ertl, H., Mingozzi, F., High, K. A., & Basner-Tschakarjan, E. (2015) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2015.29 · Cited by 84 · see citing works
The suprachoroidal space: from potential space to a space with potential
Moisseiev, E., Loewenstein, A., & Yiu, G. (2016) · Clinical Ophthalmology · doi:10.2147/opth.s89784 · Cited by 76 · see citing works
Self-citation
Predictors of Endophthalmitis after Intravitreal Injection
Stem, M. S., Rao, P., Lee, I. J., et al. (2018) · Ophthalmology Retina · doi:10.1016/j.oret.2018.09.013 · Cited by 68 · see citing works
The effect of human gene therapy for RPE65-associated Leber’s congenital amaurosis on visual function: a systematic review and meta-analysis
Wang, X., Yu, C., Tzekov, R. T., Zhu, Y., & Li, W. (2020) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-020-1304-1 · Cited by 68 · see citing works
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors
Lu, Y., & Song, S. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0909520106 · Cited by 65 · see citing works
Alterations in cytokine production following intraocular injection of soluble protein antigen: impairment in IFN-gamma and induction of TGF-beta and IL-4 production.
Kosiewicz, M. M., Alard, P., & Streilein, J. W. (1998) · PubMed · Cited by 65 · see citing works
Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-PR1. 7-hCNGB3, a recombinant AAV vector for treatment of achromatopsia
Ye, G.-J., Budzynski, E., Sonnentag, P., Nork, T. M., Miller, P. E., Sharma, A. K., Ver Hoeve, J. N., Smith, L. M., Arndt, T., Calcedo, R., et al. (2016) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2015.164 · Cited by 64 · see citing works
Prolonged Susceptibility to Antibody-mediated Neutralization for Adeno-associated Vectors Targeted to the Liver
Murphy, S. L., Li, H., Zhou, S., Schlachterman, A., & High, K. A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300334 · Cited by 62 · see citing works
AAV8 Induces Tolerance in Murine Muscle as a Result of Poor APC Transduction, T Cell Exhaustion, and Minimal MHCI Upregulation on Target Cells
Mays, L. E., Wang, L., Lin, J., Bell, P. L., Crawford, A., Wherry, E. J., & Wilson, J. M. (2013) · Molecular Therapy · doi:10.1038/mt.2013.134 · Cited by 62 · see citing works
Untitled work
(1991) · PubMed · Cited by 59 · see citing works
MyD88 Signaling in B Cells Regulates the Production of Th1-dependent Antibodies to AAV
Sudres, M., Ciré, S., Vasseur, V., Brault, L., Da Rocha, S., Boisgérault, F., Le Bec, C., Gross, D. A., Blouin, V., Ryffel, B., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.101 · Cited by 59 · 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
Host immune responses after suprachoroidal delivery of AAV8 in nonhuman primate eyes
Chung, S. H., Mollhoff, I. N., Mishra, A., Sin, T.-N., Ngo, T., Ciulla, T., Sieving, P., Thomasy, S. M., & Yiu, G. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.281 · Cited by 57 · see citing works
Self-citation
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer
Wang, D., Zhong, L., Li, M., Li, J., Tran, K., Ren, L., He, R., Xie, J., Moser, R. P., Fraser, C. K., et al. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.09.003 · Cited by 54 · see citing works
Choroidal Changes After Suprachoroidal Injection of Triamcinolone Acetonide in Eyes With Macular Edema Secondary to Retinal Vein Occlusion
Willoughby, A. S., Vuong, V. S., Cunefare, D., Farsiu, S., Noronha, G., Danis, R. P., & Yiu, G. C. (2017) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2017.11.020 · Cited by 53 · see citing works
Self-citation
Alterations in Cytokine Production Following Intraocular Injection of Soluble Protein Antigen: Impairment in IFN-γ and Induction of TGF-β and IL-4 Production
Kosiewicz, M. M., Alard, P., & Streilein, J. W. (1998) · The Journal of Immunology · doi:10.4049/jimmunol.161.10.5382 · Cited by 51 · see citing works
Soluble Fas ligand and soluble Fas in ocular fluid of patients with uveitis
Sugita, S. (2000) · British Journal of Ophthalmology · doi:10.1136/bjo.84.10.1130 · Cited by 50 · see citing works
Test–Retest Variability of Functional and Structural Parameters in Patients with Stargardt Disease Participating in the SAR422459 Gene Therapy Trial
Parker, M. A., Choi, D., Erker, L. R., Pennesi, M. E., Yang, P. G., Chegarnov, E. N., Steinkamp, P., Schlechter, C. L., Dhaenens, C., Mohand‐Saïd, S., et al. (2016) · Translational Vision Science & Technology · doi:10.1167/tvst.5.5.10 · Cited by 47 · see citing works
Regulatory and Exhausted T Cell Responses to AAV Capsid
Gernoux, G., Wilson, J. M., & Mueller, C. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.022 · Cited by 43 · see citing works
Medical and Surgical Applications for the Suprachoroidal Space
Emami-Naeini, P., & Yiu, G. (2019) · International Ophthalmology Clinics · doi:10.1097/iio.0000000000000251 · Cited by 41 · see citing works
Self-citation
Safety and Efficacy of Subretinally Administered Palucorcel for Geographic Atrophy of Age-Related Macular Degeneration
Heier, J. S., Ho, A. C., Samuel, M. A., Chang, T., Riemann, C. D., Kitchens, J. W., Slakter, J. S., Leiderman, Y. I., Spencer, R., Williams, G. A., et al. (2020) · Ophthalmology Retina · doi:10.1016/j.oret.2019.11.011 · Cited by 41 · see citing works
Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa
MacLachlan, T. K., Milton, M. N., Turner, O., Tukov, F., Choi, V. W., Penraat, J., Hanks, S. M., Vrouvlianis, J., Leehy, B., Li, H., et al. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.12.001 · Cited by 40 · see citing works
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa
Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Song, C., Timmers, A., Habecker, P. L., Pan, W., Weinstein, N. M., Swider, M., Durham, A. C., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.297 · Cited by 39 · see citing works
Treatment-Emergent Adverse Events in Gene Therapy Trials for Inherited Retinal Diseases: A Narrative Review
Nuzbrokh, Y., Kassotis, A. S., Ragi, S. D., Jauregui, R., & Tsang, S. H. (2020) · Ophthalmology and Therapy · doi:10.1007/s40123-020-00287-1 · Cited by 31 · see citing works
Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3 -Mutant Dogs
Ye, G.-J., Komáromy, A. M., Zeiss, C. J., Calcedo, R., Harman, C. D., Koehl, K. L., Stewart, G. A., Iwabe, S., Chiodo, V. A., Hauswirth, W. W., et al. (2017) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.125 · Cited by 28 · see citing works
Targeting vascular endothelial growth factor using retinal gene therapy
Chung, S. H., Frick, S. L., & Yiu, G. C. (2021) · Annals of Translational Medicine · doi:10.21037/atm-20-4417 · Cited by 25 · see citing works
Self-citation
A Subretinal Cell Delivery Method via Suprachoroidal Access in Minipigs: Safety and Surgical Outcomes
de Smet, M. D., Lynch, J. L., Dejneka, N. S., Keane, M., & Khan, I. J. (2018) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.17-22233 · Cited by 23 · see citing works
Advances in Overcoming Immune Responses following Hemophilia Gene Therapy
H. Miao, C. (2012) · Journal of Genetic Syndromes & Gene Therapy · doi:10.4172/2157-7412.s1-007 · Cited by 15 · see citing works
312. Innate Immune Response towards Adeno-Associated Viral (AAV) Vectors in Human Liver Non-Parenchymal Cells
(2010) · Molecular Therapy · doi:10.1016/s1525-0016(16)37753-x · Cited by 3 · see citing works
358. Directed Evolution of AAV for Enhanced Evasion of Human Neutralizing Antibodies
(2012) · Molecular Therapy · doi:10.1016/s1525-0016(16)36162-7 · Cited by 3 · see citing works

Flagged first, then by citations

Research works printed but not linked: 3

[37]Forthal D 2015. Functions of Antibodies, p 25– 48. In Crowe J, Boraschi D, Rappuoli R (ed), Antibodies for Infectious Diseases. ASM Press, Washington, DC.
[78]Li Q, Miller R, Han PY, Pang J, Dinculescu A, Chiodo V, Hauswirth WW. Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential. Mol Vis. 2008 9 24; 14: 1760– 9. 18836574 PMC2559816
[105]Khanani Arshad M., Kiss Szilard, Turpcu Adam, Hoang Carol, Osborne Aaron; Phase 1 Study of Intravitreal Gene Therapy ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci 2020; 61(7): 1154.

Sorted by citations

Review of gene therapies for age-related macular degeneration
Khanani, A. M., Thomas, M. J., Aziz, A. A., Weng, C. Y., Danzig, C. J., Yiu, G., Kiss, S., Waheed, N. K., & Kaiser, P. K. (2022) · Eye · doi:10.1038/s41433-021-01842-1 · Cited by 119 · see citing works
Self-citation
Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses
Ghoraba, H. H., Akhavanrezayat, A., Karaca, I., Yavari, N., Lajevardi, S., Hwang, J., Regenold, J., Matsumiya, W., Pham, B., Zaidi, M., et al. (2022) · Clinical Ophthalmology · doi:10.2147/opth.s364200 · Cited by 76 · see citing works
The degree of adeno-associated virus-induced retinal inflammation varies based on serotype and route of delivery: intravitreal, subretinal, or suprachoroidal
Wiley, L. A., Boyce, T. M., Meyering, E. E., Ochoa, D., Sheehan, K. M., Stone, E. M., Mullins, R. F., Tucker, B. A., & Han, I. C. (2023) · Human Gene Therapy · doi:10.1089/hum.2022.222 · Cited by 30 · see citing works
Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy
Purdy, R., John, M., Bray, A., Clare, A. J., Copland, D. A., Chan, Y. K., Henderson, R. H., Nerinckx, F., Leroy, B. P., Yang, P., et al. (2025) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2025.101354 · Cited by 24 · see citing works
“For example, rituximab, an anti-CD20 monoclonal antibody, could be administered around the time of first eye treatment to prevent the resulting increase in NAb titres by temporarily depleting B cell populations (Mehta et al., 2021).”
Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques
Kovacs, K. D., Ciulla, T. A., & Kiss, S. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2121646 · Cited by 20 · see citing works

Ratiometric analysis of in vivo optical coherence tomography retinal layer thicknesses for detection of changes in Alzheimer's disease
Sharma, S. N., Marsh, J. W., Tsipursky, M. S., et al. (2023) · Translational Biophotonics · doi:10.1002/tbio.202300003 · Cited by 6 · see citing works
Retinal vascular image analysis as a potential screening tool for cerebrovascular disease: a rationale based on homology between cerebral and retinal microvasculatures
Patton, N., Aslam, T. M., MacGillivray, T., Pattie, A., Deary, I. J., & Dhillon, B. (2005) · Journal of Anatomy · doi:10.1111/j.1469-7580.2005.00395.x · Cited by 811 · see citing works
Inflammation
Carton, J., Daly, R. C., & Ramani, P. (2006) · Clinical Pathology · doi:10.1093/oso/9780198569466.003.0002
Retinal vascular characteristics and the strategy to manage retinal vascular diseases
Peng, X. (2014) · Zhonghua yandibing zazhi · doi:10.3760/cma.j.issn.1005-1015.2014.05.004
Inflammation
Gopalan, C., & Kirk, E. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-823421-1.00014-7 · Cited by 4 · see citing works

Authors: Mehta, N., Robbins, D. A., & Yiu, G.
Affiliations: University of California, Davis (US)
ORCID: Mehta, Neesurg
Yiu, Glenn
Venue: International Ophthalmology Clinics
Publisher: Ovid Technologies (Wolters Kluwer Health)
Published: 2021-07
Volume: 61
Issue: 3
Pages: 151-177
ISSN: 0020-8167 (ISSN-L)
DOI: 10.1097/iio.0000000000000366
PMC: PMC8259781
OpenAlex: W3173347514
Semantic Scholar: 59bf80ae09ab3b91ab5ae9893b905e92fb8f9e83
Cited by: 33
References: 123
Citations by year: 2026: 7 · 2025: 6 · 2024: 8 · 2023: 4 · 2022: 8
FWCI: 1.57
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Locations: International Ophthalmology Clinics (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Eye Institute (R21EY031108, K08EY026101, R01EY032238)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
Health Sciences › Medicine › Ophthalmology › Ocular Diseases and Behçet’s Syndrome
Health Sciences › Medicine › Ophthalmology › Ocular Infections and Treatments
Keywords: retinal gene therapy, ocular inflammation, treatment-emergent adverse events, gene therapy safety, retinal disease
MeSH terms: Humans, Inflammation, Retina, Vision Disorders, Genetic Therapy

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

Ocular Inflammation and Treatment Emergent Adverse Events in Retinal Gene Therapy

Abstract

Integrity signals

Claims the paper makes

Claims other papers make about this one

Self-check

Cites

Cited by

Related

Record

About these checks