Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

(2019) Shouta Miyatake et al.   Molecular Therapy-Nucleic Acids

In Vitro Validation of Phosphorodiamidate Morpholino Oligomers

(2019) May T. Aung-Htut et al.   MOLECULES

Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides

(2019) May Aung-Htut et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Oligonucleotide therapy mitigates disease in Spinocerebellar Ataxia Type 3 mice

(2018) Hayley S. McLoughlin et al.   ANNALS OF NEUROLOGY

Machado-Joseph disease / Spinocerebellar ataxia type 3:lessons from disease pathogenesis and clues into therapy

(2018) Carlos Matos et al.   JOURNAL OF NEUROCHEMISTRY

Acute hepatotoxicity of 2′ fluoro-modified 5–10–5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins

(2018) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Intracerebroventricular Administration of a 2′-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain

(2018) Lodewijk J.A. Toonen et al.   Nucleic Acid Therapeutics

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

(2018) Loren L. Flynn et al.   Molecular Therapy-Nucleic Acids

Loss of the Spinocerebellar Ataxia type 3 disease protein ATXN3 alters transcription of multiple signal transduction pathways

(2018) Li Zeng et al.   PLoS One

Physiological and pathophysiological characteristics of ataxin-3 isoforms

(2018) Daniel Weishäupl et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Impact of Oligonucleotide Structure, Chemistry, and Delivery Method on In Vitro Cytotoxicity

(2017) Maja M. Janas et al.   Nucleic Acid Therapeutics

The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials

(2017) Stanley T. Crooke et al.   Nucleic Acid Therapeutics

Clinical trials of exon skipping in Duchenne muscular dystrophy

(2017) Jerry R Mendell et al.   Expert Opinion on Orphan Drugs

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

(2017) Lodewijk J.A. Toonen et al.   Molecular Therapy-Nucleic Acids

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

(2017) Lauren R. Moore et al.   Molecular Therapy-Nucleic Acids

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Effects of the enlargement of polyglutamine segments on the structure and folding of ataxin-2 and ataxin-3 proteins

(2016) Jingran Wen et al.   JOURNAL OF BIOMOLECULAR STRUCTURE & DYNAMICS

Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers

(2016) Stanley T Crooke et al.   MOLECULAR THERAPY

Splice-switching antisense oligonucleotides as therapeutic drugs

(2016) Mallory A. Havens et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3

(2016) Lodewijk J. A. Toonen et al.   Scientific Reports

The deubiquitinase ataxin-3 requires Rad23 and DnaJ-1 for its neuroprotective role in Drosophila melanogaster

(2015) Wei-Ling Tsou et al.   NEUROBIOLOGY OF DISEASE

2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF

(2015) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Toward therapeutic targets for SCA3: Insight into the role of Machado–Joseph disease protein ataxin-3 in misfolded proteins clearance

(2015) Xiaoling Li et al.   PROGRESS IN NEUROBIOLOGY

Machado Joseph disease: clinical and genetic aspects, and current treatment

(2015) Jonas Alex Morales Saute et al.   Expert Opinion on Orphan Drugs

Phosphorothioate oligonucleotides can displaceNEAT1RNA and form nuclear paraspeckle-like structures

(2014) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

(2013) Yoshitsugu Aoki et al.   HUMAN MOLECULAR GENETICS

Ataxin-3 Protein and RNA Toxicity in Spinocerebellar Ataxia Type 3: Current Insights and Emerging Therapeutic Strategies

(2013) Melvin M. Evers et al.   MOLECULAR NEUROBIOLOGY

Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon

(2013) Melvin M. Evers et al.   NEUROBIOLOGY OF DISEASE

NIH Image to ImageJ: 25 years of image analysis

(2012) Caroline A Schneider et al.   NATURE METHODS

Innate immune response gene expression profiles in central nervous system of mice infected with rabies virus

(2011) Pingsen Zhao et al.   COMPARATIVE IMMUNOLOGY MICROBIOLOGY AND INFECTIOUS DISEASES

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Machado-Joseph Disease: from first descriptions to new perspectives

(2011) Conceição Bettencourt et al.   Orphanet Journal of Rare Diseases

Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide

(2011) Melvin M. Evers et al.   PLoS One

Polyglutamine diseases: The special case of ataxin-3 and Machado–Joseph disease

(2011) Carlos A. Matos et al.   PROGRESS IN NEUROBIOLOGY

Off-Target Effects Related to the Phosphorothioate Modification of Nucleic Acids

(2010) Johannes Winkler et al.   ChemMedChem

Mouse Ataxin-3 Functional Knock-Out Model

(2010) Pawel M. Switonski et al.   NEUROMOLECULAR MEDICINE

Antiviral Activity of the Interferon-Induced Cellular Protein BST-2/Tetherin

(2009) Andrey Tokarev et al.   AIDS RESEARCH AND HUMAN RETROVIRUSES

Reversibility of symptoms in a conditional mouse model of spinocerebellar ataxia type 3

(2009) Jana Boy et al.   HUMAN MOLECULAR GENETICS

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping

(2009) Chalermchai Mitrpant et al.   MOLECULAR THERAPY

Human Splicing Finder: an online bioinformatics tool to predict splicing signals

(2009) François-Olivier Desmet et al.   NUCLEIC ACIDS RESEARCH