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Restoring vision

Nature (London), 2018-05, Vol.557 (7705), p.359-367

2018

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Gene therapy for blindness

Annual review of neuroscience, 2013-07, Vol.36 (1), p.467-488

2013

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Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa

Nature communications, 2022-12, Vol.13 (1), p.7695-7695, Article 7695

2022

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Gene therapy for inherited retinal degenerations

Comptes rendus. Biologies, 2014-03, Vol.337 (3), p.185-192

2014

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Meganuclease targeting HSV-1 protects against herpetic keratitis: Application to corneal transplants

Molecular therapy. Nucleic acids, 2022-12, Vol.30, p.511-521

2022

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Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report

BMC neurology, 2022-07, Vol.22 (1), p.257-7, Article 257

2022

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Developing gene‐independent approaches for retinal dystrophies

Acta ophthalmologica (Oxford, England), 2024-01, Vol.102 (S279), p.n/a

Sahel, José‐Alain

2024

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Gene therapy for mitochondrial diseases: Leber Hereditary Optic Neuropathy as the first candidate for a clinical trial

Comptes rendus. Biologies, 2014-03, Vol.337 (3), p.193-206

2014

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Gene therapy for the eye focus on mutation-independent approaches

Current opinion in neurology, 2015-02, Vol.28 (1), p.51-60

2015

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Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration

The Journal of clinical investigation, 2015-01, Vol.125 (1), p.105-116

2015

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The phase 3 REFLECT trial: Efficacy and safety of bilateral gene therapy for Leber hereditary optic neuropathy (LHON)

Acta ophthalmologica (Oxford, England), 2022-12, Vol.100 (S275), p.n/a

2022

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Identifying and Overcoming Challenges in Developing Effective Treatments for Usher 1B: A Workshop Report

Translational vision science & technology, 2023-02, Vol.12 (2), p.2-2

2023

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AAV-mediated gene delivery in Dp71-null mouse model with compromised barriers

Glia, 2014-03, Vol.62 (3), p.468-476

2014

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Neuroglobin Gene Therapy Prevents Optic Atrophy and Preserves Durably Visual Function in Harlequin Mice

Molecular therapy, 2014-06, Vol.22 (6), p.1096-1109

2014

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An Optical Coherence Tomography-Based Measure as an Independent Estimate of Retinal Function in Retinitis Pigmentosa

Diagnostics (Basel), 2023-11, Vol.13 (23), p.3521

2023

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Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the ND4 Gene: A Secondary Analysis of a Phase 1/2 Clinical Trial

JAMA ophthalmology, 2019-04, Vol.137 (4), p.399

2019

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Prenatal human ocular degeneration occurs in Leber's congenital amaurosis (LCA2)

The journal of gene medicine, 2002-07, Vol.4 (4), p.390-396

2002

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Gene therapy in ophthalmology: validation on cultured retinal cells and explants from postmortem human eyes

Human gene therapy, 2011-05, Vol.22 (5), p.587-593

2011

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Inherited retinal degenerations: therapeutic prospects

Biology of the Cell, 2004-05, Vol.96 (4), p.261-269

2004

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Inherited retinal degenerations: therapeutic prospects

Biology of the cell, 2004-05, Vol.96 (4), p.261-269

2004

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Current Status of Clinical Trials Design and Outcomes in Retinal Gene Therapy

Cold Spring Harbor perspectives in medicine, 2024-07, Vol.14 (7), p.a041301

2024

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Macular Dystrophies: Management and Interventions

Macular Dystrophies, 2016, p.101-116

2016

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Restoring vision

Gene therapy for blindness

Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa

Gene therapy for inherited retinal degenerations

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

Meganuclease targeting HSV-1 protects against herpetic keratitis: Application to corneal transplants

Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report

Developing gene‐independent approaches for retinal dystrophies

Gene therapy for mitochondrial diseases: Leber Hereditary Optic Neuropathy as the first candidate for a clinical trial

Gene therapy for the eye focus on mutation-independent approaches

Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration

The phase 3 REFLECT trial: Efficacy and safety of bilateral gene therapy for Leber hereditary optic neuropathy (LHON)

Identifying and Overcoming Challenges in Developing Effective Treatments for Usher 1B: A Workshop Report

AAV-mediated gene delivery in Dp71-null mouse model with compromised barriers

Neuroglobin Gene Therapy Prevents Optic Atrophy and Preserves Durably Visual Function in Harlequin Mice

An Optical Coherence Tomography-Based Measure as an Independent Estimate of Retinal Function in Retinitis Pigmentosa

Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the ND4 Gene: A Secondary Analysis of a Phase 1/2 Clinical Trial

Prenatal human ocular degeneration occurs in Leber's congenital amaurosis (LCA2)

Gene therapy in ophthalmology: validation on cultured retinal cells and explants from postmortem human eyes

Inherited retinal degenerations: therapeutic prospects

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 degenerations: therapeutic prospects

Current Status of Clinical Trials Design and Outcomes in Retinal Gene Therapy

Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation

Macular Dystrophies: Management and Interventions

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