Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy von Maguire, Albert M., Bennett, Jean, Aleman, Elena M., Leroy, Bart P., Aleman, Tomas S.

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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 t... von Bennett, Jean, Prof, Wellman, Jennifer, MSc, Marshall, Kathleen A, COT, McCague, Sarah, BA, Ashtari, Manzar, PhD, DiStefano-Pappas, Julie, BA, Elci, Okan U, PhD, Chung, Daniel C, DO, Sun, Junwei, MSc, Wright, J Fraser, PhD, Cross, Dominique R, MPH, Aravand, Puya, BA, Cyckowski, Laura L, BA, Bennicelli, Jeannette L, PhD, Mingozzi, Federico, PhD, Auricchio, Alberto, MD, Pierce, Eric A, MD, Ruggiero, Jason, MD, Leroy, Bart P, Prof, Simonelli, Francesca, Prof, High, Katherine A, MD, Maguire, Albert M, Prof

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High-resolution adaptive optics retinal imaging of cellular structure in choroideremia von Morgan, Jessica I W, Han, Grace, Klinman, Eva, Maguire, William M, Chung, Daniel C, Maguire, Albert M, Bennett, Jean

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Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2 von Testa, Francesco, MD, PhD, Maguire, Albert M., MD, Rossi, Settimio, MD, Pierce, Eric A., MD, PhD, Melillo, Paolo, PhD, Marshall, Kathleen, COT, Banfi, Sandro, MD, Surace, Enrico M., DVM, Sun, Junwei, MS, Acerra, Carmela, BA, Wright, J. Fraser, PhD, Wellman, Jennifer, MS, High, Katherine A., MD, Auricchio, Alberto, MD, Bennett, Jean, MD, PhD, Simonelli, Francesca, MD

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Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis von Maguire, Maureen G, Maguire, Albert M, Simonelli, Francesca, Pierce, Eric A, Pugh, Edward N, Mingozzi, Federico, Bennicelli, Jeannette, Banfi, Sandro, Marshall, Kathleen A, Testa, Francesco, Surace, Enrico M, Rossi, Settimio, Lyubarsky, Arkady, Arruda, Valder R, Konkle, Barbara, Stone, Edwin, Sun, Junwei, Jacobs, Jonathan, Dell'Osso, Lou, Hertle, Richard, Ma, Jian-xing, Redmond, T. Michael, Zhu, Xiaosong, Hauck, Bernd, Zelenaia, Olga, Shindler, Kenneth S, Wright, J. Fraser, Volpe, Nicholas J, McDonnell, Jennifer Wellman, Auricchio, Alberto, High, Katherine A, Bennett, Jean

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Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina von Ramachandran, Pavitra S, Lee, Vivian, Wei, Zhangyong, Song, Ji Yun, Casal, Giulia, Cronin, Therese, Willett, Keirnan, Huckfeldt, Rachel, Morgan, Jessica I W, Aleman, Tomas S, Maguire, Albert M, Bennett, Jean

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AAV9 targets cone photoreceptors in the nonhuman primate retina von Vandenberghe, Luk H, Bell, Peter, Maguire, Albert M, Xiao, Ru, Hopkins, Tim B, Grant, Rebecca, Bennett, Jean, Wilson, James M

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial von Maguire, Albert M, MD, High, Katherine A, Prof, Auricchio, Alberto, Prof, Wright, J Fraser, PhD, Pierce, Eric A, MD, Testa, Francesco, MD, Mingozzi, Federico, PhD, Bennicelli, Jeannette L, PhD, Ying, Gui-shuang, PhD, Rossi, Settimio, MD, Fulton, Ann, Prof, Marshall, Kathleen A, COT, Banfi, Sandro, MD, Chung, Daniel C, DO, Morgan, Jessica IW, PhD, Hauck, Bernd, PhD, Zelenaia, Olga, PhD, Zhu, Xiaosong, MD, Raffini, Leslie, MD, Coppieters, Frauke, PhD, De Baere, Elfride, PhD, Shindler, Kenneth S, MD, Volpe, Nicholas J, Prof, Surace, Enrico M, DVM, Acerra, Carmela, BA, Lyubarsky, Arkady, PhD, Redmond, T Michael, PhD, Stone, Edwin, Prof, Sun, Junwei, MBA, McDonnell, Jennifer Wellman, MS, Leroy, Bart P, Prof, Simonelli, Francesca, MD, Bennett, Jean, Prof

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The human visual cortex responds to gene therapy-mediated recovery of retinal function von Ashtari, Manzar, Cyckowski, Laura L, Monroe, Justin F, Marshall, Kathleen A, Chung, Daniel C, Auricchio, Alberto, Simonelli, Francesca, Leroy, Bart P, Maguire, Albert M, Shindler, Kenneth S, Bennett, Jean

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The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients von Ashtari, Manzar, PhD, Nikonova, Elena S., BA, Marshall, Kathleen A., COT, Young, Gloria J., BA, Aravand, Puya, BA, Pan, Wei, MS, Ying, Gui-shuang, PhD, Willett, Aimee E., BS, Mahmoudian, Mani, MD, Maguire, Albert M., MD, Bennett, Jean, MD, PhD

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AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models von Vasireddy, Vidyullatha, Mills, Jason A, Gaddameedi, Rajashekhar, Basner-Tschakarjan, Etiena, Kohnke, Monika, Black, Aaron D, Alexandrov, Krill, Zhou, Shangzhen, Maguire, Albert M, Chung, Daniel C, Mac, Helen, Sullivan, Lisa, Gadue, Paul, Bennicelli, Jeannette L, French, Deborah L, Bennett, Jean

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Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness von Acland, Gregory M, Aguirre, Gustavo D, Bennett, Jean, Aleman, Tomas S, Cideciyan, Artur V, Bennicelli, Jeannette, Dejneka, Nadine S, Pearce-Kelling, Susan E, Maguire, Albert M, Palczewski, Krzysztof, Hauswirth, William W, Jacobson, Samuel G

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Gene therapy restores vision in a canine model of childhood blindness von Bennett, Jean, Acland, Gregory M, Aguirre, Gustavo D, Ray, Jharna, Zhang, Qi, Aleman, Tomas S, Cideciyan, Artur V, Pearce-Kelling, Susan E, Anand, Vibha, Zeng, Yong, Maguire, Albert M, Jacobson, Samuel G, Hauswirth, William W

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Compensatory Cross-Modal Plasticity Persists After Sight Restoration von Mowad, Theresa G., Willett, Aimee E., Mahmoudian, Mani, Lipin, Mikhail, Heinecke, Armin, Maguire, Albert M., Bennett, Jean, Ashtari, Manzar

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Motion-selective areas V5/MT and MST appear resistant to deterioration in choroideremia von Silson, Edward H., Baker, Chris I., Aleman, Tomas S., Maguire, Albert M., Bennett, Jean, Ashtari, Manzar

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Identifying Photoreceptors in Blind Eyes Caused by RPE65 Mutations: Prerequisite for Human Gene Therapy Success von Jacobson, Samuel G., Aleman, Tomas S., Cideciyan, Artur V., Sumaroka, Alexander, Schwartz, Sharon B., Elizabeth A. M. Windsor, Traboulsi, Elias I., Heon, Elise, Pittler, Steven J., Milam, Ann H., Maguire, Albert M., Palczewski, Krzysztof, Stone, Edwin M., Bennett, Jean, Nathans, Jeremy

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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials von Maguire, Albert M, Russell, Stephen, Wellman, Jennifer A, Chung, Daniel C, Yu, Zi-Fan, Tillman, Amy, Wittes, Janet, Pappas, Julie, Elci, Okan, Marshall, Kathleen A, McCague, Sarah, Reichert, Hannah, Davis, Maria, Simonelli, Francesca, Leroy, Bart P, Wright, J Fraser, High, Katherine A, Bennett, Jean

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Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence von Cideciyan, Artur V., Aleman, Tomas S., Swider, Malgorzata, Schwartz, Sharon B., Steinberg, Janet D., Brucker, Alexander J., Maguire, Albert M., Bennett, Jean, Stone, Edwin M., Jacobson, Samuel G.

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Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates von Weed, Lindsey, Ammar, Michael J., Zhou, Shangzhen, Wei, Zhangyong, Serrano, Leona W., Sun, Junwei, Lee, Vivian, Maguire, Albert M., Bennett, Jean, Aleman, Tomas S.

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Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years von Maguire, Albert M, Russell, Stephen, Chung, Daniel C, Yu, Zi-Fan, Tillman, Amy, Drack, Arlene V, Simonelli, Francesca, Leroy, Bart P, Reape, Kathleen Z, High, Katherine A, Bennett, Jean

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