Preclinical Safety Evaluation of AAV2-sFLT01— A Gene Therapy for Age-related Macular Degeneration
MacLachlan, Timothy K, Lukason, Michael, Collins, Margaret, Munger, Robert, Isenberger, Elisabete, Rogers, Cindy, Malatos, Shana, DuFresne, Elizabeth, Morris, James, Calcedo, Roberto, Veres, Gabor, Scaria, Abraham, Andrews, Laura, Wadsworth, Samuel·2011·Molecular Therapy
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- What Is Next for Retinal Gene Therapy?Luk H. Vandenberghe · 2015 · Cold Spring Harbor Perspectives in Medicine
- Low risk to retina from sustained suppression of VEGFPeter A. Campochiaro · 2019 · Journal of Clinical Investigation
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- Peripapillary Intravitreal Injection Improves AAV-Mediated Retinal TransductionSanjar Batirovich Madrakhimov, Jin Young Yang, Dong Hyuck Ahn, Jung Woo Han, Tae Ho Ha, Tae Kwann Park · 2020 · Molecular Therapy — Methods & Clinical Development
- Antiangiogenic AAV2 gene therapy with a truncated form of soluble VEGFR-2 reduces the growth of choroidal neovascularization in mice after intravitreal injectionPuranen, Jooseppi, Koponen, Sanna, Nieminen, Tiina, Kanerva, Iiris, Kokki, Emmi, Toivanen, Pyry, Urtti, Arto, Ylä-Herttuala, Seppo, Ruponen, Marika · 2022 · Experimental Eye Research
- Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial DysfunctionNaomi Chadderton, Arpad Palfi, Daniel M. Maloney, Matthew Carrigan, Laura K. Finnegan, Killian S. Hanlon, Ciara Shortall, Mary O’Reilly, Peter Humphries, Lorraine Cassidy et al. · 2023 · Pharmaceutics
- Clinical Characteristics of Neovascular Age‐Related Macular Degeneration without Typical DrusenHiroyuki Kamao, Katsutoshi Goto, Kento Matsuno, Kenichi Mizukawa, Atsushi Miki, Junichi Kiryu · 2021 · Journal of Ophthalmology
- Retinal and Choroidal Neovascularization Antivascular Endothelial Growth Factor Treatments: The Role of Gene TherapySamaneh Toutounchian, Naser Ahmadbeigi, Vahid Mansouri · 2022 · Journal of Ocular Pharmacology and Therapeutics
- Breaking and Sealing Barriers in Retinal Gene TherapyJoan W. Miller, Luk H. Vandenberghe · 2018 · Molecular Therapy
- X-linked Retinoschisis and Gene TherapyAlaknanda Mishra, Paul A. Sieving · 2021 · International Ophthalmology Clinics
- Inhibition of pathological brain angiogenesis through systemic delivery of AAV vector expressing soluble FLT1Feng Shen, L Mao, Weihong Zhu, Michael T. Lawton, Peter A. Pechan, P. Colosi, Zhijian Wu, Abraham Scaria, Hua Su · 2015 · Gene Therapy
- Safety and efficacy of CRISPR-mediated genome ablation of VEGFA as a treatment for choroidal neovascularization in nonhuman primate eyesTzu-Ni Sin, Nicole Tng, Jack Dragoli, Sruthi Ramesh Kumar, Carol Villafuerte-Trisolini, Sook Hyun Chung, Lien Tu, Sophie M. Le, Jae Ho Shim, Kathryn L. Pepple et al. · 2024 · Molecular Therapy
- Morphological evaluation of retinal ganglion cells expressing the L132C/T159C ChR2 mutant transgene in young adult cynomolgus monkeysWenyao Wang, Yan Nan, Zhuo‐Hua Pan, Mingliang Pu · 2017 · Science China Life Sciences
- Today and Future of Age-Related Macular DegenerationKang Liu, Bolin Xie · 2012 · ISRN Ophthalmology
- Gene Therapies for Neovascular Age-Related Macular DegenerationPeter A. Pechan, Samuel C. Wadsworth, Abraham Scaria · 2014 · Cold Spring Harbor Perspectives in Medicine
- Importance of nonhuman primates as a model system for gene therapy development in ophthalmologyWozar, Fabian, Seitz, Immanuel, Reichel, Felix, Fischer, M. Dominik · 2022 · Klinische Monatsblätter für Augenheilkunde
- Peripheral cellular immune responses induced by subretinal adeno-associated virus gene transfer can Be restrained by the subretinal-associated immune inhibition mechanismVendomèle, Julie, Chauveau, Gaëlle Anne, Dalkara, Deniz, Galy, Anne, Fisson, Sylvain · 2024 · Human Gene Therapy
- Defects and asymmetries in the visual pathway of non-human primates with natural strabismus and amblyopiaFeng Liu, 中山大学中山眼科中心眼科学国家重点实验室, 广东省眼科学与视觉科学重点实验室, 广东 广州510060, 中国, Zhong-Hao Wang, Wanjing Huang, Ying Xu, Xuan Sang, Ruifeng Liu, Zhou-Yue Li, Ya-Lan Bi, Lei Tang et al. · 2022 · 动物学研究
- Inhibitors of AngiogenesisHildegard Büning, Ulrich Thorsten Hacker · 2016 · Advances in experimental medicine and biology
- Gene Delivery of Calreticulin Anti-Angiogenic Domain Attenuates the Development of Choroidal Neovascularization in RatsYoun‐Shen Bee, Leilei Tu, Shwu‐Jiuan Sheu, Hsiu-Chen Lin, Jia-Hua Tang, Jiang-Hui Wang, Selwyn Marc Prea, Gregory James Dusting, Deng‐Chyang Wu, Jingxiang Zhong et al. · 2017 · Human Gene Therapy
- Viral vectors engineered for gene therapyKenneth H. Lundstrom · 2023 · International review of cytology
- Anti-Angiogenic Treatment for Exudative Age-Related Macular Degeneration: New Strategies are UnderwayPatrycja M. Nowak-Sliwinska · 2012 · Current Angiogenesis
- Preventive, Diagnostic and Therapeutic Applications of Baculovirus Expression Vector SystemNeeraj Kumar, Deepak Pandey, Ashutosh Halder · 2018 · Springer eBooks
- Retinal Viral Gene Therapy: Impact of Route of Administration on Serious Adverse Events—A Systematic ReviewAubrey Berger, Ciera D. Johnson, Alan D. Marmorstein, Brittni A. Scruggs · 2025 · Clinical and Experimental Ophthalmology
- Single-dose AAV vector gene immunotherapy to treat food allergyMiguel Gonzalez-Visiedo, Xin Li, Maite Muñoz-Melero, Michael D. Kulis, Henry Daniell, David M. Markusic · 2022 · Molecular Therapy — Methods & Clinical Development
- Age-related macular degeneration (AMD): Current concepts in pathogenesis and prospects for treatmentOk Ran Shin, Yeong Hoon Kim · 2013 · Tissue Engineering and Regenerative Medicine
- Intracellular virion traffic to the endosome driven by cell type specific sialic acid receptors determines parvovirus tropismTania Calvo-López, Esther Grueso, Cristina Sánchez-Martı́nez, José María Almendral · 2023 · Frontiers in Microbiology
- Gene Therapy for Blinding Pediatric Eye DisordersAlina V. Dumitrescu, Arlene V. Drack · 2015 · Advances in Pediatrics
- Gene modification: Exploring the potential in treating kidney diseasesUbong S. Ekperikpe, Serena Zhao, Ilse Sofia Daehn · 2026 · Pharmacological Research
- MiRGD peptideticle targeted delivery of hinge-truncated soluble VEGF receptor 1 fusion protein to the retinal pigment epithelium cell line and newborn mice retinaSomayeh Piroozmand, Zahra‐Soheila Soheili, Hamid Latifi‐Navid, Shahram Samiei, Mozhgan Rezaei Kanavi, Amir Barzegar Behrooz, Saman Hosseinkhani · 2025 · International Journal of Biological Macromolecules
- [Gene therapy for hereditary ophthalmological diseases: Advances and future perspectives].Oscar Francisco Chacon-Camacho, Aline Astorga-Carballo, Juan Carlos Zenteno · 2015 · PubMed
- Gene Therapy for Retinal DiseaseJean Bennett, Albert M. Maguire · 2012 · Elsevier eBooks
- Chapter 3 - Restoring Vision to the Blind: Gene Therapy for Vision LossThe Lasker IRRF Initiative for Innovation in Vision Science The Lasker IRRF Initiative for Innovation in Vision Science · 2014 · Translational Vision Science & Technology
- Gene Therapy for Vision DisordersDeniz Dalkara, José‐Alain Sahel · 2015 · CRC Press eBooks
- Gene Therapy for Choroidal NeovascularizationJoo Yong Lee, Joon Hyung Yeo · 2020 · Springer eBooks
- Administration of Ocular Gene TherapyDavid N. Xu, Mahmood Khan, Michael A. Klufas, Allen C. Ho · 2021 · International Ophthalmology Clinics
- Ocular Gene TherapiesStylianos Michalakis, Maximilian-Joachim Gerhardt, Claudia Priglinger, Siegfried Georg Priglinger · 2021 · Springer eBooks
- New innovations for ocular gene therapyTsutomu Igarashi, Koichi Miyake, Maika Kobayashi, Kazuhisa Takahashi, Kenji Nakamoto, Takashi Okada, Hiroshi Takahashi · 2017 · Nihon Ika Daigaku Igakkai Zasshi
- An optimized gene transfection system in WERI-Rb1 cellsYing Liu, Zhigang Fan, Kang V. Li, Fei Deng, Yunfan Xiong, Meixin Liang, Jian Ge · 2017 · International Journal of Molecular Medicine
- Inhibitors of AngiogenesisHildegard Büning, Ulrich Thorsten Hacker · 2015 · Springer eBooks
- Ocular Gene TherapiesStylianos Michalakis, Maximilian-Joachim Gerhardt, Claudia Priglinger, Siegfried Georg Priglinger · 2022 · Springer eBooks
- Gene Therapy for Retinal Diseases Is Within SightCharles C. Wykoff · 2014
- Extended release anti-VEGF systems: a strategy whose time has come? Or already gone?Michael W. Stewart · 2016 · Expert Review of Ophthalmology
- Anti-Angiogenic Gene Therapy: Basic Science and Challenges for Translation into the ClinicClemens Lange, James W. Bainbridge · 2016 · Essentials in ophthalmology/Essentials ophthalmology
- Antiangiogenic Agents and Photodynamic TherapyAtul Kumar, Sumithra Mohanraj, Kavitha Duraipandi, Anuradha V. Pai · 2016 · Springer eBooks
- EXPERIMENTAL AND CLINICAL OCULAR GENE THERAPYAlexis‐Pierre Bemelmans, José‐Alain Sahel · 2019 · WORLD SCIENTIFIC (EUROPE) eBooks
- Genetic testing in translational ophthalmologyAndi Abeshi, Vincenza Precone, Tommaso Beccari, Munis Dündar, Benedetto Falsini, Matteo Bertelli · 2017 · The EuroBiotech Journal
- Terapia Génica en Degeneración Macular Relacionada con la EdadRené Alfredo Cano-Hidalgo, Carlos Quezada-Ruiz · 2013 · Highlights of Vitreoretina
- Digital Droplet PCR Method for the Quantification of AAV Transduction Efficiency in Murine RetinaIskalen Cansu Topcu Okan, Mehri Ahmadian, Yesim Tutuncu, Halit Yusuf Altay, Cavit Ağca · 2021 · Journal of Visualized Experiments
- Gene and Cell Therapy ProductsPeter K. Working · 2014
- Future therapiesPaula E. Pecen, Peter K. Kaiser · 2015 · Springer eBooks
- Cellular Immune Responses Induced by Subretinal AAV Gene Transfer can be Restrained by the Subretinal Associated Immune Inhibition MechanismJulie Vendomèle, Gaëlle Anne Chauveau, Deniz Dalkara, Anne H. M. Galy, Sylvain Fisson · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
- Role of stem cell engineering and or gene-editing technologies in eye diseases degeneration, and therapyMarwa Daghsni · 2023 · Elsevier eBooks
- Gene Expression and Editing in Mammalian Retinal Ganglion CellsNicholas M. Tran, Xiaoshu Xu, Lei S. Qi, Yang Hu · 2024 · Essentials in ophthalmology/Essentials ophthalmology
- Gene Therapy for Cornea, Conjunctiva, and Lacrimal GlandNishant R. Sinha, Rajnish Kumar, Rajiv R. Mohan · 2024 · Elsevier eBooks
- Synthesis and characterization of an injectable telechelic material for the epiretinal delivery of retinal gene therapiesJames H. Westbay, Daniel P. Bigley, Sushma Sappa, Anfisa Ayalon, Hamzah Aweidah, Lauren D. Dignam, Joseph Nissim Martel, William A. Beltran, José‐Alain Sahel, Leah C. Byrne et al. · 2026 · Journal of Materials Chemistry B
- Danggui-Shaoyao-San ameliorates metabolic dysfunction-associated steatohepatitis via suppressing hepatic macrophage NLRP3 inflammasomeQian Huang, Sa Lyu, Xin Xin, Ziming An, Qinmei Sun, Siting Gao, Hu Yiyang, Xiaojun Gou, Qin Feng · 2026 · Journal of Ethnopharmacology
- Evolution dirigée de virus adéno-associés pour un transfert de gène efficace dans le système visuelArthur Planul · 2017 · theses.fr (ABES)
- Caractérisation des réponses immunitaires systémiques induites par injection sous-rétinienne : du fondamental au transfert de gène par virus adéno-associé recombinantJulie Vendomèle · 2018 · theses.fr (ABES)
- Safety and Efficacy of cceAAV-aflibercept-Fc-YTE (F-CRG-B191): A Gene Therapy for Neovascular Age-Related Macular DegenerationJiemei Shi, Huan Xu, Hongmei Zhao, Jiajie Zhao, 陳俐卉, Ping Xu, Dan Jia, Chunhui Jiang · 2026 · Investigative Ophthalmology & Visual Science
- Intravitreal Injections in Mouse Models of Eye DiseaseAnnie L. Miller, Yashvi Bhatt, Lívia S. Carvalho · 2026 · Methods in molecular biology