Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs

Allele-Selective Inhibition of MutantHuntingtinExpression with Antisense Oligonucleotides Targeting the Expanded CAG Repeat

(2010) Keith T. Gagnon et al.   BIOCHEMISTRY

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

(2010) Jiaxin Hu et al.   CHEMISTRY & BIOLOGY

Silencing ataxin-3 mitigates degeneration in a rat model of Machado–Joseph disease: no role for wild-type ataxin-3?

(2010) Sandro Alves et al.   HUMAN MOLECULAR GENETICS

Antisense Oligonucleotides Containing Conformationally Constrained 2′,4′-(N-Methoxy)aminomethylene and 2′,4′-Aminooxymethylene and 2′-O,4′-C-Aminomethylene Bridged Nucleoside Analogues Show Improved Potency in Animal Models

(2010) Thazha P. Prakash et al.   JOURNAL OF MEDICINAL CHEMISTRY

Synthesis and Biophysical Evaluation of 2′,4′-Constrained 2′O-Methoxyethyl and 2′,4′-Constrained 2′O-Ethyl Nucleic Acid Analogues

(2010) Punit P. Seth et al.   JOURNAL OF ORGANIC CHEMISTRY

Atomic resolution structure of CAG RNA repeats: structural insights and implications for the trinucleotide repeat expansion diseases

(2010) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

RNA Interference: From Basic Research to Therapeutic Applications

(2009) Jens Kurreck   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Allele-Selective Inhibition of Mutant Huntingtin by Peptide Nucleic Acid-Peptide Conjugates, Locked Nucleic Acid, and Small Interfering RNA

(2009) Jiaxin Hu et al.   Annals of the New York Academy of Sciences

Cellular localization and allele-selective inhibition of mutant huntingtin protein by peptide nucleic acid oligomers containing the fluorescent nucleobase [bis-o-(aminoethoxy)phenyl]pyrrolocytosine

(2009) Jiaxin Hu et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

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

(2009) Jana Boy et al.   HUMAN MOLECULAR GENETICS

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

(2009) Jiaxin Hu et al.   NATURE BIOTECHNOLOGY

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

(2009) S. A. M. Mulders et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

(2009) T. M. Wheeler et al.   SCIENCE

Therapeutic gene silencing strategies for polyglutamine disorders

(2009) Janine Scholefield et al.   TRENDS IN GENETICS

Structure of an argonaute silencing complex with a seed-containing guide DNA and target RNA duplex

(2008) Yanli Wang et al.   NATURE

Mechanisms of post-transcriptional regulation by microRNAs: are the answers in sight?

(2008) Witold Filipowicz et al.   NATURE REVIEWS GENETICS

Novel Therapeutic Modalities to Address Nondrugable Protein Interaction Targets

(2008) Errol B De Souza et al.   NEUROPSYCHOPHARMACOLOGY

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

(2008) Sandro Alves et al.   PLoS One