Oligonucleotides targeting TCF4 triplet repeat expansion inhibit RNA foci and mis-splicing in Fuchs’ dystrophy

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

Antisense transcription of the myotonic dystrophy locus yields low-abundant RNAs with and without (CAG)n repeat

(2017) Anke E. E. G. Gudde et al.   RNA Biology

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33 , an Asthma-Associated Metalloprotease with Nuclear-Localized mRNA

(2017) Hannah M. Pendergraff et al.   Molecular Therapy-Nucleic Acids

RNA biology of disease-associated microsatellite repeat expansions

(2017) Kushal J. Rohilla et al.   Acta Neuropathologica Communications

MicroRNA-29b Overexpression Decreases Extracellular Matrix mRNA and Protein Production in Human Corneal Endothelial Cells

(2016) Tetsuya Toyono et al.   CORNEA

Five-Year Graft Survival Comparing Descemet Stripping Automated Endothelial Keratoplasty and Penetrating Keratoplasty

(2016) Marcus Ang et al.   OPHTHALMOLOGY

RNA Toxicity and Missplicing in the Common Eye Disease Fuchs Endothelial Corneal Dystrophy

(2015) Jintang Du et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy

(2015) Agnieszka Wojtkowiak-Szlachcic et al.   NUCLEIC ACIDS RESEARCH

Correlation of Severity of Fuchs Endothelial Corneal Dystrophy With Triplet Repeat Expansion inTCF4

(2015) Ahmed Z. Soliman et al.   JAMA Ophthalmology

Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs

(2014) Jiaxin Hu et al.   BIOCHEMISTRY

Transcript profile of cellular senescence-related genes in Fuchs endothelial corneal dystrophy

(2014) Mario Matthaei et al.   EXPERIMENTAL EYE RESEARCH

Aganirsen Antisense Oligonucleotide Eye Drops Inhibit Keratitis-Induced Corneal Neovascularization and Reduce Need for Transplantation

(2014) Claus Cursiefen et al.   OPHTHALMOLOGY

Antisense transcripts of the expanded C9ORF72 hexanucleotide repeat form nuclear RNA foci and undergo repeat-associated non-ATG translation in c9FTD/ALS

(2013) Tania F. Gendron et al.   ACTA NEUROPATHOLOGICA

Locked nucleic acid: tighter is different

(2013) Jonathan K. Watts   CHEMICAL COMMUNICATIONS

RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia

(2013) T. Zu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Common Trinucleotide Repeat Expansion within the Transcription Factor 4 (TCF4, E2-2) Gene Predicts Fuchs Corneal Dystrophy

(2012) Eric D. Wieben et al.   PLoS One

Characterization of cellular senescence mechanisms in human corneal endothelial cells

(2011) Angela N. Sheerin et al.   AGING CELL

Evidence of Oxidative Stress in the Pathogenesis of Fuchs Endothelial Corneal Dystrophy

(2010) Ula V. Jurkunas et al.   AMERICAN JOURNAL OF PATHOLOGY

Allele-Selective Inhibition of Huntingtin Expression by Switching to an miRNA-like RNAi Mechanism

(2010) Jiaxin Hu et al.   CHEMISTRY & BIOLOGY

Descemet's Stripping Endothelial Keratoplasty

(2010) Marianne O. Price et al.   OPHTHALMOLOGY

Non-ATG-initiated translation directed by microsatellite expansions

(2010) T. Zu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs

(2009) Jiaxin Hu et al.   NATURE BIOTECHNOLOGY

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

(2009) C. A. Stein et al.   NUCLEIC ACIDS RESEARCH

Descemet's Stripping Endothelial Keratoplasty: Safety and Outcomes

(2009) W. Barry Lee et al.   OPHTHALMOLOGY