Pharmacotherapy of retinal disease with visual cycle modulators

Gene therapy for inherited retinal and optic nerve degenerations

(2017) Nicholas A. Moore et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Novel therapeutics for Stargardt disease

(2017) Louise J. Lu et al.   GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Delayed Rod-Mediated Dark Adaptation Is a Functional Biomarker for Incident Early Age-Related Macular Degeneration

(2016) Cynthia Owsley et al.   OPHTHALMOLOGY

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

PHASE II, RANDOMIZED, PLACEBO-CONTROLLED, 90-DAY STUDY OF EMIXUSTAT HYDROCHLORIDE IN GEOGRAPHIC ATROPHY ASSOCIATED WITH DRY AGE-RELATED MACULAR DEGENERATION

(2015) Pravin U. Dugel et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

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

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

Geographic Atrophy

(2014) Frank G. Holz et al.   OPHTHALMOLOGY

Similar molecules spatially correlate with lipofuscin and N-retinylidene-N-retinylethanolamine in the mouse but not in the human retinal pigment epithelium

(2013) Zsolt Ablonczy et al.   ARCHIVES OF BIOCHEMISTRY AND BIOPHYSICS

Lutein + Zeaxanthin and Omega-3 Fatty Acids for Age-Related Macular Degeneration

(2013)   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Treatment With 9-cisβ-Carotene–Rich Powder in Patients With Retinitis Pigmentosa

(2013) Ygal Rotenstreich et al.   JAMA Ophthalmology

Vitamin A Metabolism in Rod and Cone Visual Cycles

(2012) John C. Saari   Annual Review of Nutrition

INVESTIGATION OF ORAL FENRETINIDE FOR TREATMENT OF GEOGRAPHIC ATROPHY IN AGE-RELATED MACULAR DEGENERATION

(2012) Nathan L. Mata et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

Complement System Dysregulation and Inflammation in the Retinal Pigment Epithelium of a Mouse Model for Stargardt Macular Degeneration

(2011) Roxana A. Radu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Primary amines protect against retinal degeneration in mouse models of retinopathies

(2011) Akiko Maeda et al.   Nature Chemical Biology

SAFETY AND EFFECT ON ROD FUNCTION OF ACU-4429, A NOVEL SMALL-MOLECULE VISUAL CYCLE MODULATOR

(2011) Ryo Kubota et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

Deuterium Enrichment of Vitamin A at the C20 Position Slows the Formation of Detrimental Vitamin A Dimers in Wild-type Rodents

(2010) Yardana Kaufman et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

The Cone-specific visual cycle

(2010) Jin-Shan Wang et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate

(2009) T. Maeda et al.   HUMAN MOLECULAR GENETICS

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