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The new landscape of retinal gene therapy

American journal of medical genetics. Part C, Seminars in medical genetics, 2020-09, Vol.184 (3), p.846-859

2020

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Detailed Clinical Phenotype and Molecular Genetic Findings in CLN3-Associated Isolated Retinal Degeneration

JAMA ophthalmology, 2017-07, Vol.135 (7), p.749

2017

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Expanding the phenotypic and genotypic spectrum of patients with HGSNAT -related retinopathy

Ophthalmic genetics, 2024-04, Vol.45 (2), p.167-174

2024

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The Role of ERK1/2 Activation in Sarpogrelate-Mediated Neuroprotection

Investigative ophthalmology & visual science, 2018-01, Vol.59 (1), p.462-471

2018

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Vitelliform maculopathy in MELAS syndrome

American journal of ophthalmology case reports, 2023-06, Vol.30, p.101842, Article 101842

2023

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Retinal Neuroprotective Effects of Flibanserin, an FDA-Approved Dual Serotonin Receptor Agonist-Antagonist

PloS one, 2016-07, Vol.11 (7), p.e0159776-e0159776

2016

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Bilateral ocular injuries with globe perforation and retinitis sclopetaria from birdshot shotgun: A case report and review of the literature

Trauma case reports, 2020-08, Vol.28, p.100329, Article 100329

2020

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Gene Therapy Trial Update: A Primer for Vitreoretinal Specialists

Ophthalmic surgery, lasers & imaging retina, 2016-01, Vol.47 (1), p.6-12

2016

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Stargardt Disease: Gene Therapy Strategies for ABCA4

International ophthalmology clinics, 2021-10, Vol.61 (4), p.157-165

2021

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X-Linked Retinoschisis

Cold Spring Harbor perspectives in medicine, 2023-09, Vol.13 (9), p.a041288

2023

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Granulicatella adiacens, an unusual causative agent in chronic dacryocystitis

Journal of ophthalmic inflammation and infection, 2015-04, Vol.5 (1), p.12-12, Article 12

2015

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Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

Human molecular genetics, 2011-12, Vol.20 (23), p.4569-4581

2011

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Molecular and Clinical Findings in Patients With Knobloch Syndrome

JAMA ophthalmology, 2016-07, Vol.134 (7), p.753

2016

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Improved Rod Sensitivity as Assessed by Two-Color Dark-Adapted Perimetry in Patients With RPE65-Related Retinopathy Treated With Voretigene Neparvovec-rzyl

Translational vision science & technology, 2023-04, Vol.12 (4), p.17-17

2023

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Perimacular Atrophy Following Voretigene Neparvovec-Rzyl Treatment in the Setting of Previous Contralateral Eye Treatment With a Different Viral Vector

Translational vision science & technology, 2024-06, Vol.13 (6), p.11

2024

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Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model

Human molecular genetics, 2015-02, Vol.24 (3), p.670-684

2015

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Retinal gene therapy: current progress and future prospects

Expert review of ophthalmology, 2015-06, Vol.10 (3), p.281-299

2015

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Spectrum of ocular manifestations in cobalamin C and cobalamin A types of methylmalonic acidemia

Ophthalmic genetics, 2016-12, Vol.37 (4), p.404-414

2016

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Retracting Globe: Enophthalmos and Retraction Due to an Accessory Extraocular Muscle

Journal of neuro-ophthalmology, 2015-06, Vol.35 (2), p.227-229

2015

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Efficacy of Aflibercept in Bevacizumab-Resistant Macular Edema in Adult-Onset Coats Disease

Journal of vitreoretinal diseases (Print), 2019-11, Vol.3 (6), p.480-484

2019

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Vitelliform maculopathy in MELAS syndrome

American journal of ophthalmology case reports, 2023, Vol.30, p.101842-101842

2023

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Bilateral ocular injuries with globe perforation and retinitis sclopetaria from birdshot shotgun: A case report and review of the literature

Trauma case reports, 2020, Vol.28, p.100329-100329

2020

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Spectrum of ocular manifestations in cobalamin C and cobalamin A types of methylmalonic acidemia

Ophthalmic Genetics, 2016, Vol.37 (4), p.404-414

2016

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Inherited Retinal Disease Panels-Caveat Emptor-Truly Know Your Inherited Retinal Disease Panel

Retina (Philadelphia, Pa.), 2022-01, Vol.42 (1), p.1-3

2022

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The new landscape of retinal gene therapy

Detailed Clinical Phenotype and Molecular Genetic Findings in CLN3-Associated Isolated Retinal Degeneration

Expanding the phenotypic and genotypic spectrum of patients with HGSNAT -related retinopathy

The Role of ERK1/2 Activation in Sarpogrelate-Mediated Neuroprotection

Vitelliform maculopathy in MELAS syndrome

Retinal Neuroprotective Effects of Flibanserin, an FDA-Approved Dual Serotonin Receptor Agonist-Antagonist

Bilateral ocular injuries with globe perforation and retinitis sclopetaria from birdshot shotgun: A case report and review of the literature

Gene Therapy Trial Update: A Primer for Vitreoretinal Specialists

Stargardt Disease: Gene Therapy Strategies for ABCA4

X-Linked Retinoschisis

Granulicatella adiacens, an unusual causative agent in chronic dacryocystitis

Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

Molecular and Clinical Findings in Patients With Knobloch Syndrome

Improved Rod Sensitivity as Assessed by Two-Color Dark-Adapted Perimetry in Patients With RPE65-Related Retinopathy Treated With Voretigene Neparvovec-rzyl

Perimacular Atrophy Following Voretigene Neparvovec-Rzyl Treatment in the Setting of Previous Contralateral Eye Treatment With a Different Viral Vector

Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model

Retinal gene therapy: current progress and future prospects

Spectrum of ocular manifestations in cobalamin C and cobalamin A types of methylmalonic acidemia

Retracting Globe: Enophthalmos and Retraction Due to an Accessory Extraocular Muscle

Efficacy of Aflibercept in Bevacizumab-Resistant Macular Edema in Adult-Onset Coats Disease

Vitelliform maculopathy in MELAS syndrome

Bilateral ocular injuries with globe perforation and retinitis sclopetaria from birdshot shotgun: A case report and review of the literature

Spectrum of ocular manifestations in cobalamin C and cobalamin A types of methylmalonic acidemia

Three‐Year Safety Results of SAR422459 (EIAV‐ABCA4) Gene Therapy in Patients With ABCA4‐Associated Stargardt Disease: An Open‐Label Dose‐Escalation Phase I/IIa Clinical Trial, Cohorts 1‐5

Inherited Retinal Disease Panels-Caveat Emptor-Truly Know Your Inherited Retinal Disease Panel

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