Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

Exploring the Effect of Sequence Length and Composition on Allele-Selective Inhibition of Human Huntingtin Expression by Single-Stranded Silencing RNAs

(2014) Jiaxin Hu et al.   Nucleic Acid Therapeutics

Allele-Selective Inhibition of Expression of Huntingtin and Ataxin-3 by RNA Duplexes Containing Unlocked Nucleic Acid Substitutions

(2013) Yuichiro Aiba et al.   BIOCHEMISTRY

Treatment of Huntington Disease

(2013) Aleksandar Videnovic   Current Treatment Options in Neurology

CAG size-specific risk estimates for intermediate allele repeat instability in Huntington disease

(2013) Alicia Semaka et al.   JOURNAL OF MEDICAL GENETICS

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Multisource ascertainment of Huntington disease in Canada: Prevalence and population at risk

(2013) Emily R. Fisher et al.   MOVEMENT DISORDERS

ss-siRNAs allele selectively inhibit ataxin-3 expression: multiple mechanisms for an alternative gene silencing strategy

(2013) Jing Liu et al.   NUCLEIC ACIDS RESEARCH

RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression

(2013) J. Liu et al.   NUCLEIC ACIDS RESEARCH

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

(2013) Michael E. Østergaard et al.   NUCLEIC ACIDS RESEARCH

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

A fully humanized transgenic mouse model of Huntington disease

(2012) Amber L. Southwell et al.   HUMAN MOLECULAR GENETICS

Assessing unintended hybridization-induced biological effects of oligonucleotides

(2012) Morten Lindow et al.   NATURE BIOTECHNOLOGY

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

TricycloDNA-modified oligo-2′-deoxyribonucleotides reduce scavenger receptor B1 mRNA in hepatic and extra-hepatic tissues—a comparative study of oligonucleotide length, design and chemistry

(2012) Sue Murray et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

(2012) Amber L. Southwell et al.   TRENDS IN MOLECULAR MEDICINE

Silencing disease genes in the laboratory and the clinic

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

Development of biomarkers for Huntington's disease

(2011) David W Weir et al.   LANCET NEUROLOGY

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

(2011) Jeffrey B Carroll et al.   MOLECULAR THERAPY

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

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

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

Tetrabenazine: the first approved drug for the treatment of chorea in US patients with Huntington disease

(2010) Samuel Frank   Neuropsychiatric Disease and Treatment

CAG Expansion in the Huntington Disease Gene Is Associated with a Specific and Targetable Predisposing Haplogroup

(2009) Simon C. Warby et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

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

Multiple alphaII-spectrin breakdown products distinguish calpain and caspase dominated necrotic and apoptotic cell death pathways

(2009) Zhiqun Zhang et al.   APOPTOSIS

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference

(2009) Maria Stella Lombardi et al.   EXPERIMENTAL NEUROLOGY

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

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

(2009) Jiaxin Hu et al.   NATURE BIOTECHNOLOGY

Improvement of RNA secondary structure prediction using RNase H cleavage and randomized oligonucleotides

(2009) A. D. Kauffmann et al.   NUCLEIC ACIDS RESEARCH

Identification and Allele-Specific Silencing of the Mutant Huntingtin Allele in Huntington's Disease Patient-Derived Fibroblasts

(2008) P.H.J. van Bilsen et al.   HUMAN GENE THERAPY

Short Antisense Oligonucleotides with Novel 2′−4′ Conformationaly Restricted Nucleoside Analogues Show Improved Potency without Increased Toxicity in Animals

(2008) Punit P. Seth et al.   JOURNAL OF MEDICINAL CHEMISTRY

Allele-specific silencing of mutant Huntington’s disease gene

(2008) Yu Zhang et al.   JOURNAL OF NEUROCHEMISTRY