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Measuring Rod-and Cone-Photoreceptor-Specific Vision in Inherited Retinal Diseases Using a Commercial Perimeter Wu, Vivian; Roman, Alejandro J.; Galsterer, Emma L.; Ansari, Georg; Erdinest, Inbar; Righetti, Giulia; Viarbitskaya, Iryna; Russell, Robert C.; Kim, Rebecca J.; Charlier, Jacques; Pfau, Kristina; Stingl, Krunoslav; Banin, Eyal; Pfau, Maximilian; Stingl, Katarina; Aleman, Tomas S.; Cideciyan, Artur, V Share Save
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Retinal Degeneration Associated With Biallelic RDH12 Variants: Longitudinal Evaluation of Retinal Structure and Visual Function in Pediatric Patients Aleman, Tomas S.; Roman, Alejandro J.; Uyhazi, Katherine E.; Jiang, Yu You; Bedoukian, Emma C.; Sumaroka, Alexander; Wu, Vivian; Swider, Malgorzata; Viarbitskaya, Iryna; Russell, Robert C.; Shagena, Elizabeth O.; Santos, Arlene J.; Serrano, Leona W.; Parchinski, Kelsey M.; Kim, Rebecca J.; Weber, Mariejel L.; Garafalo, Alexandra, V; Thompson, Dorothy A.; Maguire, Albert M.; Bennett, Jean; Scoles, Drew H.; O'Neil, Erin C.; Morgan, Jessica I. W.; Cideciyan, Artur, V Share Save
Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations (vol 24, 102409, 2021) Jacobson, Samuel G.; Cideciyan, Artur, V; Ho, Allen C.; Peshenko, Igor, V; Garafalo, Alexandra, V; Roman, Alejandro J.; Sumaroka, Alexander; Wu, Vivian; Krishnan, Arun K.; Sheplock, Rebecca; Cheang, Bee-Lin; Davidson, Vanessa; O'Riordan, Catherine R.; Boye, Sanford L.; Dizhoor, Alexander M.; Boye, Shannon E. Share Save
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Treatment Strategy With Gene Editing for Late-Onset Retinal Degeneration Caused by a Founder Variant in C1QTNF5 Li, Randa T. H.; Roman, Alejandro J.; Sumaroka, Alexander; Stanton, Chloe M.; Swider, Malgorzata; Garafalo, Alexandra V.; Heon, Elise; Vincent, Ajoy; Wright, Alan F.; Megaw, Roly; Aleman, Tomas S.; Browning, Andrew C.; Dhillon, Baljean; Cideciyan, Artur V. Share Save
Progression of Stargardt Disease as Determined by Fundus Autofluorescence Over a 24-Month Period (ProgStar Report No. 17) Strauss, Rupert W.; Ho, Alexander; Jha, Anamika; Fujinami, Kaoru; Michaelides, Michel; Cideciyan, Artur V.; Audo, Isabelle; Birch, David G.; Sadda, Srinivas; Ip, Michael; West, Sheila; Schonbach, Etienne M.; Kong, Xiangrong; Scholl, Hendrik P. N. Share Save
Photoreceptor Function and Structure in Autosomal Dominant Vitelliform Macular Dystrophy Caused by BEST1 Mutations Cideciyan, Artur V.; Jacobson, Samuel G.; Swider, Malgorzata; Sumaroka, Alexander; Sheplock, Rebecca; Krishnan, Arun K.; Garafalo, Alexandra V.; Guziewicz, Karina E.; Aguirre, Gustavo D.; Beltran, William A.; Heon, Elise Share Save
The landscape of submicroscopic structural variants at the OPN1LW/OPN1MW gene cluster on Xq28 underlying blue cone monochromacy Wissinger, Bernd; Baumann, Britta; Buena-Atienza, Elena; Ravesh, Zeinab; Cideciyan, Artur, V; Stingl, Katarina; Audo, Isabelle; Meunier, Isabelle; Bocquet, Beatrice; Traboulsi, Elias, I; Hardcastle, Alison J.; Gardner, Jessica C.; Michaelides, Michel; Branham, Kari E.; Rosenberg, Thomas; Andreasson, Sten; Dollfus, Helene; Birch, David; Vincent, Andrea L.; Martorell, Loreto; Mora, Jaume Catala; Kellner, Ulrich; Ruther, Klaus; Lorenz, Birgit; Preising, Markus N.; Manfredini, Emanuela; Zarate, Yuri A.; Vijzelaar, Raymon; Zrenner, Eberhart; Jacobson, Samuel G.; Kohl, Susanne Share Save
Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial Cideciyan, Artur, V; Jacobson, Samuel G.; Ho, Allen C.; Krishnan, Arun K.; Roman, Alejandro J.; Garafalo, Alexandra, V; Wu, Vivian; Swider, Malgorzata; Sumaroka, Alexander; Van Cauwenbergh, Caroline; Russell, Stephen R.; Drack, Arlene, V; Leroy, Bart P.; Schwartz, Michael R.; Girach, Aniz Share Save
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial Russell, Stephen R.; Drack, Arlene, V; Cideciyan, Artur, V; Jacobson, Samuel G.; Leroy, Bart P.; Van Cauwenbergh, Caroline; Ho, Allen C.; Dumitrescu, Alina, V; Han, Ian C.; Martin, Mitchell; Pfeifer, Wanda L.; Sohn, Elliott H.; Walshire, Jean; Garafalo, Alexandra, V; Krishnan, Arun K.; Powers, Christian A.; Sumaroka, Alexander; Roman, Alejandro J.; Vanhonsebrouck, Eva; Jones, Eltanara; Nerinckx, Fanny; De Zaeytijd, Julie; Collin, Rob W. J.; Hoyng, Carel; Adamson, Peter; Cheetham, Michael E.; Schwartz, Michael R.; den Hollander, Wilhelmina; Asmus, Friedrich; Platenburg, Gerard; Rodman, David; Girach, Aniz Share Save
Longitudinal Changes in Scotopic and Mesopic Macular Function as Assessed with Microperimetry in Patients With Stargardt Disease: SMART Study Report No. 2 Kong, Xiangrong; Ibrahim-Ahmed, Mohamed; Bittencourt, Millena G.; Strauss, Rupert W.; Birch, David G.; Cideciyan, Artur, V; Ervin, Ann-Margaret; Ho, Alexander; Sunness, Janet S.; Audo, Isabelle S.; Michaelides, Michel; Zrenner, Eberhart; Sadda, SriniVas; Ip, Michael S.; West, Sheila; Scholl, Hendrik P. N. Share Save
Longitudinal Changes of Fixation Stability and Location Within 24 Months in Stargardt Disease: ProgStar Report No. 16 Schoenbach, Etienne M.; Strauss, Rupert W.; Cattaneo, Marco E. G., V; Fujinami, Kaoru; Birch, David G.; Cideciyan, Artur, V; Sunness, Janet S.; Zrenner, Eberhart; Sadda, SriniVas R.; Scholl, Hendrik P. N. 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
The Progression of Stargardt Disease Using Volumetric Hill of Vision Analyses Over 24 Months: ProgStar Report No.15 Schonbach, Etienne M.; Janeschitz-Kriegl, Lucas; Strauss, Rupert W.; Cattaneo, Marco E. G., V; Fujinami, Kaoru; Birch, David G.; Cideciyan, Artur, V; Sunness, Janet S.; Weleber, Richard G.; Ip, Michael S.; Sadda, SriniVas R.; Scholl, Hendrik P. N. 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
Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report Cideciyan, Artur V.; Jacobson, Samuel G.; Ho, Allen C.; Garafalo, Alexandra V.; Roman, Alejandro J.; Sumaroka, Alexander; Krishnan, Arun K.; Swider, Malgorzata; Schwartz, Michael R.; Girach, Aniz Share Save
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