Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia.

Autor: Ross M; Koret School of Veterinary Medicine, Hebrew University of Jerusalem, Rehovot, Israel., Obolensky A; Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel., Averbukh E; Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel., Desrosiers M; Department of Therapeutics, Institut de la Vision, Paris, France., Ezra-Elia R; Koret School of Veterinary Medicine, Hebrew University of Jerusalem, Rehovot, Israel., Honig H; Department of Animal Science, ARO, The Volcani Center, Rishon LeZion, Israel., Yamin E; Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel., Rosov A; Department of Animal Science, ARO, The Volcani Center, Rishon LeZion, Israel., Dvir H; Department of Animal Science, ARO, The Volcani Center, Rishon LeZion, Israel., Gootwine E; Department of Animal Science, ARO, The Volcani Center, Rishon LeZion, Israel., Banin E; Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Jerusalem, Israel., Dalkara D; Department of Therapeutics, Institut de la Vision, Paris, France., Ofri R; Koret School of Veterinary Medicine, Hebrew University of Jerusalem, Rehovot, Israel. ron.ofri@mail.huji.ac.il.

Publikováno v: Gene therapy [Gene Ther] 2022 Nov; Vol. 29 (10-11), pp. 624-635. Date of Electronic Publication: 2021 Dec 02.

Evaluation of Retinal Structure and Visual Function in Blue Cone Monochromacy to Develop Clinical Endpoints for L-opsin Gene Therapy.

Autor: Cideciyan, Artur V.¹ (AUTHOR) aroman@pennmedicine.upenn.edu, Roman, Alejandro J.¹ (AUTHOR) raymond.warner@pennmedicine.upenn.edu, Warner, Raymond L.¹ (AUTHOR) asumarok@pennmedicine.upenn.edu, Sumaroka, Alexander¹ (AUTHOR) vivian.wu@pennmedicine.upenn.edu, Wu, Vivian¹ (AUTHOR) yuyou.jiang@pennmedicine.upenn.edu, Jiang, Yu Y.¹ (AUTHOR) mswider@pennmedicine.upenn.edu, Swider, Malgorzata¹ (AUTHOR) garafalo@pennmedicine.upenn.edu, Garafalo, Alexandra V.¹ (AUTHOR) iryna.viarbitskaya@pennmedicine.upenn.edu, Viarbitskaya, Iryna¹ (AUTHOR) robert.russell2@pennmedicine.upenn.edu, Russell, Robert C.¹ (AUTHOR) jwmorgan@pennmedicine.upenn.edu, Kohl, Susanne² (AUTHOR) susanne.kohl@med.uni-tuebingen.de, Wissinger, Bernd² (AUTHOR) bernd.wissinger@med.uni-tuebingen.de, Ripamonti, Caterina³ (AUTHOR) c.ripamonti@crsltd.com, Barbur, John L.⁴ (AUTHOR) j.l.barbur@city.ac.uk, Bach, Michael⁵ (AUTHOR) bach@uni-freiburg.de, Carroll, Joseph⁶ (AUTHOR) jcarroll@mcw.edu, Morgan, Jessica I. W.¹ (AUTHOR) aleman@pennmedicine.upenn.edu, Aleman, Tomas S.¹ (AUTHOR)

Publikováno v: International Journal of Molecular Sciences. Oct2024, Vol. 25 Issue 19, p10639. 23p.

Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep.

Autor: Gootwine E; 1 Agricultural Research Organization, The Volcani Center , Rishon LeZion, Israel ., Ofri R; 2 Koret School of Veterinary Medicine, Hebrew University of Jerusalem , Rehovot, Israel ., Banin E; 3 Department of Ophthalmology, Hadassah-Hebrew University Medical Center , Jerusalem, Israel ., Obolensky A; 3 Department of Ophthalmology, Hadassah-Hebrew University Medical Center , Jerusalem, Israel ., Averbukh E; 3 Department of Ophthalmology, Hadassah-Hebrew University Medical Center , Jerusalem, Israel ., Ezra-Elia R; 2 Koret School of Veterinary Medicine, Hebrew University of Jerusalem , Rehovot, Israel ., Ross M; 2 Koret School of Veterinary Medicine, Hebrew University of Jerusalem , Rehovot, Israel ., Honig H; 1 Agricultural Research Organization, The Volcani Center , Rishon LeZion, Israel ., Rosov A; 1 Agricultural Research Organization, The Volcani Center , Rishon LeZion, Israel ., Yamin E; 3 Department of Ophthalmology, Hadassah-Hebrew University Medical Center , Jerusalem, Israel ., Ye GJ; 4 Applied Genetic Technologies Corporation (AGTC) , Alachua, Florida., Knop DR; 4 Applied Genetic Technologies Corporation (AGTC) , Alachua, Florida., Robinson PM; 4 Applied Genetic Technologies Corporation (AGTC) , Alachua, Florida., Chulay JD; 4 Applied Genetic Technologies Corporation (AGTC) , Alachua, Florida., Shearman MS; 4 Applied Genetic Technologies Corporation (AGTC) , Alachua, Florida.

Publikováno v: Human gene therapy. Clinical development [Hum Gene Ther Clin Dev] 2017 Jun; Vol. 28 (2), pp. 96-107. Date of Electronic Publication: 2017 May 05.

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Seeing the Future: A Review of Ocular Therapy.

Autor: Whalen, Maiya¹ (AUTHOR), Akula, Monica² (AUTHOR), McNamee, Shannon M.² (AUTHOR), DeAngelis, Margaret M.³ (AUTHOR), Haider, Neena B.^2,4 (AUTHOR) neena_haider@hms.harvard.edu

Publikováno v: Bioengineering (Basel). Feb2024, Vol. 11 Issue 2, p179. 40p.