RNA therapeutics in ophthalmology - translation to clinical trials

Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy

(2020) Kasia Dzierlega et al.   GENE THERAPY

The powerful world of antisense oligonucleotides: From bench to bedside

(2020) Anaïs M. Quemener et al.   Wiley Interdisciplinary Reviews-RNA

Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases

(2020) Kanmin Xue et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

(2020) Karishma Dhuri et al.   Journal of Clinical Medicine

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Leber Congenital Amaurosis Associated with Mutations in CEP290 , Clinical Phenotype, and Natural History in Preparation for Trials of Novel Therapies

(2018) Leo Sheck et al.   OPHTHALMOLOGY

The molecular and cellular basis of rhodopsin retinitis pigmentosa reveals potential strategies for therapy

(2018) Dimitra Athanasiou et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Special Issue Introduction: Inherited Retinal Disease: Novel Candidate Genes, Genotype–Phenotype Correlations, and Inheritance Models

(2018) Frans Cremers et al.   Genes

Development of targeted siRNA nanocomplexes to prevent fibrosis in experimental glaucoma filtration surgery

(2018) Owen Fernando et al.   MOLECULAR THERAPY

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

(2018) Kalyan Dulla et al.   Molecular Therapy-Nucleic Acids

Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

(2018) Artur V. Cideciyan et al.   NATURE MEDICINE

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

(2017) Carlo Rinaldi et al.   Nature Reviews Neurology

The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market

(2017) Ricardo Titze-de-Almeida et al.   PHARMACEUTICAL RESEARCH

First-in-human phase I study of ISTH0036, an antisense oligonucleotide selectively targeting transforming growth factor beta 2 (TGF-β2), in subjects with open-angle glaucoma undergoing glaucoma filtration surgery

(2017) Norbert Pfeiffer et al.   PLoS One

Efficacy and Safety of 0.1% Cyclosporine a Cationic Emulsion in the Treatment of Severe Dry Eye Disease: A Multicenter Randomized Trial

(2016) Andrea Leonardi et al.   EUROPEAN JOURNAL OF OPHTHALMOLOGY

Receptor-targeted liposome-peptide-siRNA nanoparticles represent an efficient delivery system for MRTF silencing in conjunctival fibrosis

(2016) Cynthia Yu-Wai-Man et al.   Scientific Reports

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

(2015) Melissa A. Kotterman et al.   Annual Review of Biomedical Engineering

Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities

(2015) Pranav Mathur et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Review and update on the molecular basis of Leber congenital amaurosis

(2015) Oscar Francisco Chacon-Camacho   World Journal of Clinical Cases

The effect of the common c.2299delG mutation in USH2A on RNA splicing

(2014) Eva Lenassi et al.   EXPERIMENTAL EYE RESEARCH

Geographic Atrophy

(2014) Frank G. Holz et al.   OPHTHALMOLOGY

Small-interfering RNAs (siRNAs) as a promising tool for ocular therapy

(2013) A Guzman-Aranguez et al.   BRITISH JOURNAL OF PHARMACOLOGY

Mitomycin C versus5-fluorouracil as an adjunctive treatment for trabeculectomy: a meta-analysis of randomized clinical trials

(2013) Lígia I De Fendi et al.   CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Using the Early Postoperative Intraocular Pressure to Predict Pressure Control After a Trabeculectomy

(2013) Satoshi Okimoto et al.   JOURNAL OF GLAUCOMA

Phase I Clinical Trial of SYL040012, a Small Interfering RNA Targeting β-Adrenergic Receptor 2, for Lowering Intraocular Pressure

(2013) Javier Moreno-Montañés et al.   MOLECULAR THERAPY

Seven new loci associated with age-related macular degeneration

(2013) Lars G Fritsche et al.   NATURE GENETICS

Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients

(2012) Q D Nguyen et al.   EYE

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

Evaluation of the siRNA PF-04523655 versus Ranibizumab for the Treatment of Neovascular Age-related Macular Degeneration (MONET Study)

(2012) Quan Dong Nguyen et al.   OPHTHALMOLOGY

siRNA-optimized Modifications for Enhanced In Vivo Activity

(2012) Denise M Kenski et al.   Molecular Therapy-Nucleic Acids

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

High-Dose siRNAs Upregulate Mouse Eri-1 at both Transcription and Posttranscription Levels

(2011) Yingnan Bian et al.   PLoS One

RNAi-Based Treatment for Neovascular Age-Related Macular Degeneration by Sirna-027

(2010) Peter K. Kaiser et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

The pathogenic role of transforming growth factor-β2 in glaucomatous damage to the optic nerve head

(2010) Rudolf Fuchshofer   EXPERIMENTAL EYE RESEARCH

Influence of Race and Age on Aqueous Humor Levels of Transforming Growth Factor-Beta 2 in Glaucomatous and Nonglaucomatous Eyes

(2010) Rupal H. Trivedi et al.   JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS

Two-Year Outcomes of the Ranibizumab for Edema of the mAcula in Diabetes (READ-2) Study

(2010) Quan Dong Nguyen et al.   OPHTHALMOLOGY

Factors Associated with Improvement and Worsening of Visual Acuity 2 Years after Focal/Grid Photocoagulation for Diabetic Macular Edema

(2010) Lloyd Paul Aiello et al.   OPHTHALMOLOGY

Prevalence of Age-related Macular Degeneration in Old Persons: Age, Gene/Environment Susceptibility Reykjavik Study

(2010) Fridbert Jonasson et al.   OPHTHALMOLOGY

Change in Area of Geographic Atrophy in the Age-Related Eye Disease Study

(2009)   ARCHIVES OF OPHTHALMOLOGY

Origins and Mechanisms of miRNAs and siRNAs

(2009) Richard W. Carthew et al.   CELL

The RNA-induced Silencing Complex: A Versatile Gene-silencing Machine

(2009) Ashley J. Pratt et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A three-dimensional view of the molecular machinery of RNA interference

(2009) Martin Jinek et al.   NATURE

Primary End Point (Six Months) Results of the Ranibizumab for Edema of the mAcula in Diabetes (READ-2) Study

(2009) Quan Dong Nguyen et al.   OPHTHALMOLOGY

Small interfering RNA-induced TLR3 activation inhibits blood and lymphatic vessel growth

(2009) W. G. Cho et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

COMBINATION THERAPY USING THE SMALL INTERFERING RNA BEVASIRANIB

(2009) LAWRENCE SINGERMAN   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

Delivery Vehicles for Small Interfering RNA In Vivo

(2008) Antonin R. de Fougerolles   HUMAN GENE THERAPY

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

(2008) Mark E. Kleinman et al.   NATURE

Oxidative damage–induced inflammation initiates age-related macular degeneration

(2008) Joe G Hollyfield et al.   NATURE MEDICINE

Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides

(2008) R. Juliano et al.   NUCLEIC ACIDS RESEARCH

Chemical Modification of siRNAs for In Vivo Use

(2008) Mark A. Behlke   OLIGONUCLEOTIDES