Emerging therapies for inherited retinal degeneration

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

(2016) Benjamin Bakondi et al.   MOLECULAR THERAPY

The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Studies

(2016) Rupert W. Strauss et al.   OPHTHALMOLOGY

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

(2016) Richard G. Weleber et al.   OPHTHALMOLOGY

Is There Excess Oxidative Stress and Damage in Eyes of Patients with Retinitis Pigmentosa?

(2015) Peter A. Campochiaro et al.   ANTIOXIDANTS & REDOX SIGNALING

Synthetic 3D diamond-based electrodes for flexible retinal neuroprostheses: Model, production and in vivo biocompatibility

(2015) Amel Bendali et al.   BIOMATERIALS

Assessment of estimated retinal atrophy progression in Stargardt macular dystrophy using spectral-domain optical coherence tomography

(2015) Rupert W Strauss et al.   BRITISH JOURNAL OF OPHTHALMOLOGY

Rod-Derived Cone Viability Factor Promotes Cone Survival by Stimulating Aerobic Glycolysis

(2015) Najate Aït-Ali et al.   CELL

Personalized therapeutic strategies for patients with retinitis pigmentosa

(2015) Andrew Zheng et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Thioredoxin rod-derived cone viability factor protects against photooxidative retinal damage

(2015) G. Elachouri et al.   FREE RADICAL BIOLOGY AND MEDICINE

Advancing Therapeutic Strategies for Inherited Retinal Degeneration: Recommendations From the Monaciano Symposium

(2015) D. A. Thompson et al.   INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE

Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies

(2015) Steven D Schwartz et al.   LANCET

Photovoltaic restoration of sight with high visual acuity

(2015) Henri Lorach et al.   NATURE MEDICINE

Optogenetics and the future of neuroscience

(2015) Edward S Boyden   NATURE NEUROSCIENCE

Optogenetics: 10 years of microbial opsins in neuroscience

(2015) Karl Deisseroth   NATURE NEUROSCIENCE

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

(2015) Samuel G. Jacobson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Rescue of the Stargardt phenotype inAbca4knockout mice through inhibition of vitamin A dimerization

(2015) Peter Charbel Issa et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transplantation of human embryonic stem cell-derived retinal tissue in two primate models of retinal degeneration

(2015) Hiroshi Shirai et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179

(2015) Koji M. Nishiguchi et al.   Nature Communications

Effectiveness, safety and costs of orphan drugs: an evidence-based review

(2015) Igho J Onakpoya et al.   BMJ Open

Treatment of Macular Degeneration Using Embryonic Stem Cell-Derived Retinal Pigment Epithelium: Preliminary Results in Asian Patients

(2015) Won Kyung Song et al.   Stem Cell Reports

Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)

(2015) Hendrik P. N. Scholl et al.   PLoS One

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

(2014) Leah C. Byrne et al.   JOURNAL OF CLINICAL INVESTIGATION

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Gene therapy arrives at the macula

(2014) Hendrik P N Scholl et al.   LANCET

Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial

(2014) Robert K Koenekoop et al.   LANCET

Independent optical excitation of distinct neural populations

(2014) Nathan C Klapoetke et al.   NATURE METHODS

Noninvasive optical inhibition with a red-shifted microbial rhodopsin

(2014) Amy S Chuong et al.   NATURE NEUROSCIENCE

Proposed Lexicon for Anatomic Landmarks in Normal Posterior Segment Spectral-Domain Optical Coherence Tomography

(2014) Giovanni Staurenghi et al.   OPHTHALMOLOGY

Daylight Vision Repair by Cell Transplantation

(2014) Tiago Santos-Ferreira et al.   STEM CELLS

The cost-effectiveness of the Argus II retinal prosthesis in Retinitis Pigmentosa patients

(2014) Anil Vaidya et al.   BMC Ophthalmology

A comparison of the causes of blindness certifications in England and Wales in working age adults (16–64 years), 1999–2000 with 2009–2010

(2014) Gerald Liew et al.   BMJ Open

Randomized Trial of Ciliary Neurotrophic Factor Delivered by Encapsulated Cell Intraocular Implants for Retinitis Pigmentosa

(2013) David G. Birch et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Gene Therapy for Blindness

(2013) José-Alain Sahel et al.   Annual Review of Neuroscience

The Argus II epiretinal prosthesis system allows letter and word reading and long-term function in patients with profound vision loss

(2013) Lyndon da Cruz et al.   BRITISH JOURNAL OF OPHTHALMOLOGY

Chemistry of the Retinoid (Visual) Cycle

(2013) Philip D. Kiser et al.   CHEMICAL REVIEWS

Adaptive Optics Retinal Imaging – Clinical Opportunities and Challenges

(2013) Joseph Carroll et al.   CURRENT EYE RESEARCH

ReaChR: a red-shifted variant of channelrhodopsin enables deep transcranial optogenetic excitation

(2013) John Y Lin et al.   NATURE NEUROSCIENCE

Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

(2013) Francesco Testa et al.   OPHTHALMOLOGY

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

(2013) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation

(2013) M. S. Singh et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Spectral-Domain Optical Coherence Tomography Measures of Outer Segment Layer Progression in Patients With X-Linked Retinitis Pigmentosa

(2013) David G. Birch et al.   JAMA Ophthalmology

GPR179 Is Required for Depolarizing Bipolar Cell Function and Is Mutated in Autosomal-Recessive Complete Congenital Stationary Night Blindness

(2012) Neal S. Peachey et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Generating an iPSC Bank for HLA-Matched Tissue Transplantation Based on Known Donor and Recipient HLA Types

(2012) Craig J. Taylor et al.   Cell Stem Cell

Neuroprotective Strategies for the Treatment of Inherited Photoreceptor Degeneration

(2012) D. Trifunovic et al.   CURRENT MOLECULAR MEDICINE

Restoration of vision after transplantation of photoreceptors

(2012) R. A. Pearson et al.   NATURE

Interim Results from the International Trial of Second Sight's Visual Prosthesis

(2012) Mark S. Humayun et al.   OPHTHALMOLOGY

Outer Segment Formation of Transplanted Photoreceptor Precursor Cells

(2012) Dominic Eberle et al.   PLoS One

Repair of the degenerate retina by photoreceptor transplantation

(2012) A. C. Barber et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Optogenetic therapy for retinitis pigmentosa

(2011) V Busskamp et al.   GENE THERAPY

Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

(2011) Sophia Millington-Ward et al.   MOLECULAR THERAPY

Self-organizing optic-cup morphogenesis in three-dimensional culture

(2011) Mototsugu Eiraku et al.   NATURE

Primary amines protect against retinal degeneration in mouse models of retinopathies

(2011) Akiko Maeda et al.   Nature Chemical Biology

Ultra light-sensitive and fast neuronal activation with the Ca2+-permeable channelrhodopsin CatCh

(2011) Sonja Kleinlogel et al.   NATURE NEUROSCIENCE

Transplantation of Adult Mouse iPS Cell-Derived Photoreceptor Precursors Restores Retinal Structure and Function in Degenerative Mice

(2011) Budd A. Tucker et al.   PLoS One

CNTF and retina

(2011) Rong Wen et al.   PROGRESS IN RETINAL AND EYE RESEARCH

A hybrid bioorganic interface for neuronal photoactivation

(2011) Diego Ghezzi et al.   Nature Communications

Cone and rod photoreceptor transplantation in models of the childhood retinopathy Leber congenital amaurosis using flow-sorted Crx-positive donor cells

(2010) J. Lakowski et al.   HUMAN MOLECULAR GENETICS

C20-D3-vitamin A Slows Lipofuscin Accumulation and Electrophysiological Retinal Degeneration in a Mouse Model of Stargardt Disease

(2010) Li Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina

(2010) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Generation, Purification and Transplantation of Photoreceptors Derived from Human Induced Pluripotent Stem Cells

(2010) Deepak A. Lamba et al.   PLoS One

Structure-Function Correlation of the Human Central Retina

(2010) Peter Charbel Issa et al.   PLoS One

Subretinal electronic chips allow blind patients to read letters and combine them to words

(2010) E. Zrenner et al.   PROCEEDINGS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES

Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa

(2010) V. Busskamp et al.   SCIENCE

Rod-Derived Cone Viability Factor for Treating Blinding Diseases: From Clinic to Redox Signaling

(2010) T. Leveillard et al.   Science Translational Medicine

Transplantation of Human Embryonic Stem Cell-Derived Photoreceptors Restores Some Visual Function in Crx-Deficient Mice

(2009) Deepak A. Lamba et al.   Cell Stem Cell

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Improved Focalization of Electrical Microstimulation Using Microelectrode Arrays: A Modeling Study

(2009) Sébastien Joucla et al.   PLoS One

Prospects for retinal gene replacement therapy

(2009) Alexander J. Smith et al.   TRENDS IN GENETICS

Vision Improvement in Retinal Degeneration Patients by Implantation of Retina Together with Retinal Pigment Epithelium

(2008) Norman D. Radtke et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration

(2008) Pamela S Lagali et al.   NATURE NEUROSCIENCE

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA