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Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa Occelli, Laurence M.; Zobel, Lena; Stoddard, Jonathan; Wagner, Johanna; Pasmanter, Nathaniel; Querubin, Janice; Renner, Lauren M.; Reynaga, Rene; Winkler, Paige A.; Sun, Kelian; Marinho, Luis Felipe L. P.; O'Riordan, Catherine R.; Frederick, Amy; Lauer, Andreas; Tsang, Stephen H.; Hauswirth, William W.; McGill, Trevor J.; Neuringer, Martha; Michalakis, Stylianos; Petersen-Jones, Simon M. Share Save
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Neuroprotective Gene Therapy by Overexpression of the Transcription Factor MAX in Rat Models of Glaucomatous Neurodegeneration Lani-Louzada, Rafael; Marra, Camila; Dias, Mariana Santana; de Araujo, Victor Guedes; Abreu, Carla Andreia; Ribas, Vinicius Toledo; Adesse, Daniel; Allodi, Silvana; Chiodo, Vince; Hauswirth, William; Petrs-Silva, Hilda; Linden, Rafael Share Save
Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis (vol 29, pg 2456, 2021) Aguirre, Gustavo D.; V. Cideciyan, Artur; Dufour, Valerie L.; Ripolles-Garcia, Ana; Sudharsan, Raghavi; Swider, Malgorzata; Nikonov, Roman; Iwabe, Simone; Boye, Sanford L.; Hauswirth, William W.; Jacobson, Samuel G.; Beltran, William A. Share Save
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Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis Aguirre, Gustavo D.; Cideciyan, Artur, V; Dufour, Valerie L.; Ripolles-Garcia, Ana; Sudharsan, Raghavi; Swider, Malgorzata; Nikonov, Roman; Iwabe, Simone; Boye, Sanford L.; Hauswirth, William W.; Jacobson, Samuel G.; Beltran, William A. Share Save
CNGB1-related rod-cone dystrophy: A mutation review and update Nassisi, Marco; Smirnov, Vasily M.; Solis Hernandez, Cyntia; Mohand-Said, Saddek; Condroyer, Christel; Antonio, Aline; Kuehlewein, Laura; Kempf, Melanie; Kohl, Susanne; Wissinger, Bernd; Nasser, Fadi; Ragi, Sara D.; Wang, Nan-Kai; Sparrow, Janet R.; Greenstein, Vivienne C.; Michalakis, Stylianos; Mahroo, Omar A.; Ba-Abbad, Rola; Michaelides, Michel; Webster, Andrew R.; Degli Esposti, Simona; Saffren, Brooke; Capasso, Jenina; Levin, Alex; Hauswirth, William W.; Dhaenens, Claire-Marie; Defoort-Dhellemmes, Sabine; Tsang, Stephen H.; Zrenner, Eberhart; Sahel, Jose-Alain; Petersen-Jones, Simon M.; Zeitz, Christina; Audo, Isabelle Share Save
Interplay between cell-adhesion molecules governs synaptic wiring of cone photoreceptors Cao, Yan; Wang, Yuchen; Dunn, Henry A.; Orlandi, Cesare; Shultz, Nicole; Kamasawa, Naomi; Fitzpatrick, David; Li, Wei; Zeitz, Christina; Hauswirth, William; Martemyanov, Kirill A. Share Save
XIAP Protects Retinal Ganglion Cells in the Mutant ND4 Mouse Model of Leber Hereditary Optic Neuropathy Wassmer, Sarah J.; De Repentigny, Yves; Sheppard, Derek; Lagali, Pamela S.; Fang, Lijun; Coupland, Stuart G.; Kothary, Rashmi; Guy, John; Hauswirth, William W.; Tsilfidis, Catherine Share Save
Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy Gardiner, Kristin L.; Cideciyan, Artur V.; Swider, Malgorzata; Dufour, Valerie L.; Sumaroka, Alexander; Komaromy, Andras M.; Hauswirth, William W.; Iwabe, Simone; Jacobson, Samuel G.; Beltran, William A.; Aguirre, Gustavo D. Share Save
Clarin-1 expression in adult mouse and human retina highlights a role of Muller glia in Usher syndrome Xu, Lei; Bolch, Susan N.; Santiago, Clayton P.; Dyka, Frank M.; Akil, Omar; Lobanova, Ekaterina S.; Wang, Yuchen; Martemyanov, Kirill A.; Hauswirth, William W.; Smith, W. Clay; Handa, James T.; Blackshaw, Seth; Ash, John D.; Dinculescu, Astra Share Save
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Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model Akil, Omar; Dyka, Frank; Calvet, Charlotte; Emptoz, Alice; Lahlou, Ghizlene; Nouaille, Sylvie; de Monvel, Jacques Boutet; Hardelin, Jean-Pierre; Hauswirth, William W.; Avan, Paul; Petit, Christine; Safieddine, Saaid; Lustig, Lawrence R. Share Save
C3-targeted retinal gene therapy limits neurodegeneration onset and progression in age-related mouse glaucoma Bosco, Alejandra; Anderson, Sarah; Breen, Kevin; Romero, Cesar; Steele, Michael; Chiodo, Vince; Boyle, Sanford; Hauswirth, William; Tomlinson, Stephen; Vetter, Monica Share Save
Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma 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. Share Save
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector Cideciyan, Artur V.; Sudharsan, Raghavi; Dufour, Valerie L.; Massengill, Michael T.; Iwabe, Simone; Swider, Malgorzata; Lisi, Brianna; Sumaroka, Alexander; Marinho, Luis Felipe; Appelbaum, Tatyana; Rossmiller, Brian; Hauswirth, William W.; Jacobson, Samuel G.; Lewin, Alfred S.; Aguirre, Gustavo D.; Beltran, William A. Share Save
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Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy (vol 8, 2017) Zhang, Yuxin; Deng, Wen-Tao; Du, Wei; Zhu, Ping; Li, Jie; Xu, Fan; Sun, Jingfen; Gerstner, Cecilia D.; Baehr, Wolfgang; Boye, Sanford L.; Zhao, Chen; Hauswirth, William W.; Pang, Ji-jing Share Save
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