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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Proceedings of the National Academy of Sciences - PNAS, 2013-02, Vol.110 (6), p.1984-1984

2013

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Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

Proceedings of the National Academy of Sciences - PNAS, 2012-02, Vol.109 (6), p.2132-2137

2012

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Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

Proceedings of the National Academy of Sciences - PNAS, 2015-10, Vol.112 (43), p.E5844-E5853

2015

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Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis

Molecular therapy, 2021-08, Vol.29 (8), p.2456-2468

2021

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Canine retina has a primate fovea-like bouquet of cone photoreceptors which is affected by inherited macular degenerations

PloS one, 2014-03, Vol.9 (3), p.e90390-e90390

2014

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Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects

PloS one, 2013-10, Vol.8 (10), p.e75666-e75666

2013

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Photochemical Restoration of Light Sensitivity in the Degenerated Canine Retina

Pharmaceutics, 2022-12, Vol.14 (12), p.2711

2022

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Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Proceedings of the National Academy of Sciences - PNAS, 2018-09, Vol.115 (36), p.E8547-E8556

2018

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Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy

Molecular therapy, 2020-01, Vol.28 (1), p.266-278

2020

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BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure

Proceedings of the National Academy of Sciences - PNAS, 2018-03, Vol.115 (12), p.E2839-E2848

2018

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Quantitative and qualitative characterization of retinal dystrophies in canine models of inherited retinal diseases using spectral domain optical coherence tomography (SD-OCT)

Experimental eye research, 2022-07, Vol.220, p.109106-109106, Article 109106

2022

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Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3 -congenital stationary night blindness

Proceedings of the National Academy of Sciences - PNAS, 2022-03, Vol.119 (13), p.e2117038119-e2117038119

2022

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Consensus guidelines for nomenclature of companion animal inherited retinal disorders

Veterinary ophthalmology, 2024-02

2024

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Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Molecular therapy, 2017-08, Vol.25 (8), p.1866-1880

2017

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Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis

Molecular therapy, 2021-12, Vol.29 (12), p.3528-3528

2021

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Overlap of abnormal photoreceptor development and progressive degeneration in Leber congenital amaurosis caused by NPHP5 mutation

Human molecular genetics, 2016-10, Vol.25 (19), p.4211-4226

2016

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Assessment of visual function and retinal structure following acute light exposure in the light sensitive T4R rhodopsin mutant dog

Experimental eye research, 2016-05, Vol.146, p.341-353

2016

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Assessment of Rod, Cone, and Intrinsically Photosensitive Retinal Ganglion Cell Contributions to the Canine Chromatic Pupillary Response

Investigative ophthalmology & visual science, 2017-01, Vol.58 (1), p.65-78

2017

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Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology

Scientific reports, 2017-10, Vol.7 (1), p.12823-15, Article 12823

2017

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Focal/multifocal and geographic retinal dysplasia in the dog—In vivo retinal microanatomy analyses

Veterinary ophthalmology, 2020-03, Vol.23 (2), p.292-304

2020

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Biomechanical properties and correlation with collagen solubility profile in the posterior sclera of canine eyes with an ADAMTS10 mutation

Investigative ophthalmology & visual science, 2013-04, Vol.54 (4), p.2685-2695

2013

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Author Correction: Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology

Scientific reports, 2018-08, Vol.8 (1), p.13058-1, Article 13058

2018

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Tolerability of Topical Tocilizumab Eyedrops in Dogs: A Pilot Study

Journal of ocular pharmacology and therapeutics, 2017-09, Vol.33 (7), p.519

2017

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Exclusion of RPGRIP1 ins44 from primary causal association with early-onset cone-rod dystrophy in dogs

Investigative ophthalmology & visual science, 2012-08, Vol.53 (9), p.5486-5501

2012

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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis

Canine retina has a primate fovea-like bouquet of cone photoreceptors which is affected by inherited macular degenerations

Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects

Photochemical Restoration of Light Sensitivity in the Degenerated Canine Retina

Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy

BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure

Quantitative and qualitative characterization of retinal dystrophies in canine models of inherited retinal diseases using spectral domain optical coherence tomography (SD-OCT)

Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3 -congenital stationary night blindness

Consensus guidelines for nomenclature of companion animal inherited retinal disorders

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis

Overlap of abnormal photoreceptor development and progressive degeneration in Leber congenital amaurosis caused by NPHP5 mutation

Assessment of visual function and retinal structure following acute light exposure in the light sensitive T4R rhodopsin mutant dog

Assessment of Rod, Cone, and Intrinsically Photosensitive Retinal Ganglion Cell Contributions to the Canine Chromatic Pupillary Response

Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology

Focal/multifocal and geographic retinal dysplasia in the dog—In vivo retinal microanatomy analyses

Biomechanical properties and correlation with collagen solubility profile in the posterior sclera of canine eyes with an ADAMTS10 mutation

Author Correction: Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology

Tolerability of Topical Tocilizumab Eyedrops in Dogs: A Pilot Study

Exclusion of RPGRIP1 ins44 from primary causal association with early-onset cone-rod dystrophy in dogs

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