Current Clinical Applications of In Vivo Gene Therapy with AAVs von Mendell, Jerry R., Al-Zaidy, Samiah A., Rodino-Klapac, Louise R., Goodspeed, Kimberly, Gray, Steven J., Kay, Christine N., Boye, Sanford L., Boye, Shannon E., George, Lindsey A., Salabarria, Stephanie, Corti, Manuela, Byrne, Barry J., Tremblay, Jacques P.

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A Comprehensive Review of Retinal Gene Therapy von Boye, Shannon E, Boye, Sanford L, Lewin, Alfred S, Hauswirth, William W

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Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model von Yu, Hong, Koilkonda, Rajeshwari D, Chou, Tsung-Han, Porciatti, Vittorio, Ozdemir, Sacide S, Chiodo, Vince, Boye, Sanford L, Boye, Shannon E, Hauswirth, William W, Lewin, Alfred S, Guy, John

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SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration von Sasaki, Yo, Kakita, Hiroki, Kubota, Shunsuke, Sene, Abdoulaye, Lee, Tae Jun, Ban, Norimitsu, Dong, Zhenyu, Lin, Joseph B., Boye, Sanford L., DiAntonio, Aaron, Boye, Shannon E., Apte, Rajendra S., Milbrandt, Jeffrey

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Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors von Boye, Sanford L, Bennett, Antonette, Scalabrino, Miranda L, McCullough, K Tyler, Van Vliet, Kim, Choudhury, Shreyasi, Ruan, Qing, Peterson, James, Agbandje-McKenna, Mavis, Boye, Shannon E

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Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma von Bosco, Alejandra, Anderson, Sarah R., Breen, Kevin T., Romero, Cesar O., Steele, Michael R., Chiodo, Vince A., Boye, Sanford L., Hauswirth, William W., Tomlinson, Stephen, Vetter, Monica L.

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SubILM Injection of AAV for Gene Delivery to the Retina von Gamlin, Paul D., Alexander, John J., Boye, Sanford L., Witherspoon, C. Douglas, Boye, Shannon E.

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Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat von Bogner, Barbara, Boye, Sanford L, Min, Seok Hong, Peterson, James J, Ruan, Qing, Zhang, Zhonghong, Reitsamer, Herbert A, Hauswirth, William W, Boye, Shannon E

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Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse von Boye, Shannon E, Boye, Sanford L, Pang, Jijing, Ryals, Renee, Everhart, Drew, Umino, Yumiko, Neeley, Andy W, Besharse, Joseph, Barlow, Robert, Hauswirth, William W

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Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina von Boye, Shannon E, Alexander, John J, Witherspoon, C Douglas, Boye, Sanford L, Peterson, James J, Clark, Mark E, Sandefer, Kristen J, Girkin, Chris A, Hauswirth, William W, Gamlin, Paul D

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AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia von Pang, Ji-jing, Deng, Wen-Tao, Dai, Xufeng, Lei, Bo, Everhart, Drew, Umino, Yumiko, Li, Jie, Zhang, Keqing, Mao, Song, Boye, Sanford L, Liu, Li, Chiodo, Vince A, Liu, Xuan, Shi, Wei, Tao, Ye, Chang, Bo, Hauswirth, William W

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Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness von Scalabrino, Miranda L, Boye, Sanford L, Fransen, Kathryn M H, Noel, Jennifer M, Dyka, Frank M, Min, Seok Hong, Ruan, Qing, De Leeuw, Charles N, Simpson, Elizabeth M, Gregg, Ronald G, McCall, Maureen A, Peachey, Neal S, Boye, Shannon E

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Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects von Guziewicz, Karina E, Zangerl, Barbara, Komáromy, András M, Iwabe, Simone, Chiodo, Vincent A, Boye, Sanford L, Hauswirth, William W, Beltran, William A, Aguirre, Gustavo D

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Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa von Beltran, William A., Cideciyan, Artur V., Lewin, Alfred S., Iwabe, Simone, Khanna, Hemant, Sumaroka, Alexander, Chiodo, Vince A., Fajardo, Diego S., Román, Alejandro J., Deng, Wen-Tao, Swider, Malgorzata, Alemán, Tomas S., Boye, Sanford L., Genini, Sem, Swaroop, Anand, Hauswirth, William W., Jacobson, Samuel G., Aguirre, Gustavo D.

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Virally delivered Channelrhodopsin-2 Safely and Effectively Restores Visual Function in Multiple Mouse Models of Blindness von Doroudchi, M Mehdi, Greenberg, Kenneth P, Liu, Jianwen, Silka, Kimberly A, Boyden, Edward S, Lockridge, Jennifer A, Arman, A Cyrus, Janani, Ramesh, Boye, Shannon E, Boye, Sanford L, Gordon, Gabriel M, Matteo, Benjamin C, Sampath, Alapakkam P, Hauswirth, William W, Horsager, Alan

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Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis von Aguirre, Gustavo D., Cideciyan, Artur V., Dufour, Valérie L., Ripolles-García, Ana, Sudharsan, Raghavi, Swider, Malgorzata, Nikonov, Roman, Iwabe, Simone, Boye, Sanford L., Hauswirth, William W., Jacobson, Samuel G., Beltran, William A.

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Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease von Beltran, William A., Cideciyan, Artur V., Iwabe, Simone, Swider, Malgorzata, Kosyk, Mychajlo S., McDaid, Kendra, Martynyuk, Inna, Ying, Gui-Shuang, Shaffer, James, Deng, Wen-Tao, Boye, Sanford L., Lewin, Alfred S., Hauswirth, William W., Jacobson, Samuel G., Aguirre, Gustavo D.

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Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa von Pang, Ji-jing, Dai, Xufeng, Boye, Shannon E, Barone, Ilaria, Boye, Sanford L, Mao, Song, Everhart, Drew, Dinculescu, Astra, Liu, Li, Umino, Yumiko, Lei, Bo, Chang, Bo, Barlow, Robert, Strettoi, Enrica, Hauswirth, William W

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Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach von Petersen-Jones, Simon M, Occelli, Laurence M, Winkler, Paige A, Lee, Winston, Sparrow, Janet R, Tsukikawa, Mai, Boye, Sanford L, Chiodo, Vince, Capasso, Jenina E, Becirovic, Elvir, Schön, Christian, Seeliger, Mathias W, Levin, Alex V, Michalakis, Stylianos, Hauswirth, William W, Tsang, Stephen H

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ELOVL4-Mediated Production of Very Long-Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability von Kady, Nermin M, Liu, Xuwen, Lydic, Todd A, Syed, Meesum H, Navitskaya, Svetlana, Wang, Qi, Hammer, Sandra S, O'Reilly, Sandra, Huang, Chao, Seregin, Sergey S, Amalfitano, Andrea, Chiodo, Vince A, Boye, Sanford L, Hauswirth, William W, Antonetti, David A, Busik, Julia V

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