Published in Neuron on June 21, 2012
Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration. Proc Natl Acad Sci U S A (2013) 2.95
Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs. Nat Neurosci (2012) 2.93
Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion. Sci Transl Med (2013) 2.89
Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell (2012) 2.83
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol (2013) 2.43
HDAC4 reduction: a novel therapeutic strategy to target cytoplasmic huntingtin and ameliorate neurodegeneration. PLoS Biol (2013) 1.96
Towards a therapy for Angelman syndrome by targeting a long non-coding RNA. Nature (2014) 1.87
Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease. Nat Med (2014) 1.67
Rhes deletion is neuroprotective in the 3-nitropropionic acid model of Huntington's disease. J Neurosci (2013) 1.55
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Mechanisms of toxicity in C9FTLD/ALS. Acta Neuropathol (2014) 1.35
Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs. Neuron (2016) 1.35
Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy. Cell Rep (2014) 1.32
Huntington's disease: underlying molecular mechanisms and emerging concepts. Trends Biochem Sci (2013) 1.31
Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models. Nat Genet (2012) 1.31
Antisense reduction of tau in adult mice protects against seizures. J Neurosci (2013) 1.29
The unstable repeats--three evolving faces of neurological disease. Neuron (2013) 1.26
Antisense-based therapy for the treatment of spinal muscular atrophy. J Cell Biol (2012) 1.25
Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. Nucleic Acids Res (2013) 1.23
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Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway. Nucleic Acids Res (2012) 1.20
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Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice. Proc Natl Acad Sci U S A (2012) 1.06
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Deletion or Inhibition of the Oxygen Sensor PHD1 Protects against Ischemic Stroke via Reprogramming of Neuronal Metabolism. Cell Metab (2016) 1.05
Direct intraventricular delivery of drugs to the rodent central nervous system. J Vis Exp (2013) 1.01
A fully humanized transgenic mouse model of Huntington disease. Hum Mol Genet (2012) 1.01
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Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease. Hum Gene Ther (2014) 0.96
Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing. Hum Mol Genet (2013) 0.95
RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression. Nucleic Acids Res (2013) 0.94
CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases. Mol Neurodegener (2015) 0.94
Allele-selective inhibition of expression of huntingtin and ataxin-3 by RNA duplexes containing unlocked nucleic acid substitutions. Biochemistry (2013) 0.93
Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain. Mol Ther Nucleic Acids (2013) 0.92
Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice. Mol Ther (2014) 0.92
Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Mol Ther Nucleic Acids (2015) 0.91
Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides. Open Biol (2012) 0.91
Oligonucleotide-based strategies to combat polyglutamine diseases. Nucleic Acids Res (2014) 0.90
Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs. Nucleic Acid Ther (2014) 0.90
"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease. Neuron (2012) 0.90
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients. PLoS One (2014) 0.90
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Antisense therapy in neurology. J Pers Med (2013) 0.87
In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2. Eur J Hum Genet (2012) 0.87
Huntingtin is required for normal excitatory synapse development in cortical and striatal circuits. J Neurosci (2014) 0.87
Spinal muscular atrophy: journeying from bench to bedside. Neurotherapeutics (2014) 0.87
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther (2015) 0.87
Intrabodies as neuroprotective therapeutics. Neurotherapeutics (2013) 0.87
Antisense technology: an emerging platform for cardiovascular disease therapeutics. J Cardiovasc Transl Res (2013) 0.87
Mouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II. Mol Neurobiol (2012) 0.86
Transgenic animal models for study of the pathogenesis of Huntington's disease and therapy. Drug Des Devel Ther (2015) 0.86
Suppressing angiotensinogen synthesis attenuates kidney cyst formation in a Pkd1 mouse model. FASEB J (2015) 0.86
Selective expression of mutant huntingtin during development recapitulates characteristic features of Huntington's disease. Proc Natl Acad Sci U S A (2016) 0.85
Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells. PLoS One (2014) 0.85
Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing. Mol Ther (2016) 0.85
Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels. J Clin Invest (2016) 0.85
Bidirectional control of postsynaptic density-95 (PSD-95) clustering by Huntingtin. J Biol Chem (2013) 0.84
Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry. Mol Ther (2015) 0.83
Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain. Nucleic Acids Res (2014) 0.83
Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases. Nucleic Acid Ther (2014) 0.83
Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain. Front Genet (2016) 0.82
Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease. Mol Ther Nucleic Acids (2016) 0.82
Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression. Sci Rep (2015) 0.81
TRiC subunits enhance BDNF axonal transport and rescue striatal atrophy in Huntington's disease. Proc Natl Acad Sci U S A (2016) 0.81
Small non-coding RNAs add complexity to the RNA pathogenic mechanisms in trinucleotide repeat expansion diseases. Front Mol Neurosci (2013) 0.81
Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies. Mol Neurobiol (2013) 0.81
Mutant huntingtin gene-dose impacts on aggregate deposition, DARPP32 expression and neuroinflammation in HdhQ150 mice. PLoS One (2013) 0.81
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo. Mol Ther (2017) 0.80
Translational research in Huntington's disease: opening up for disease modifying treatment. Transl Neurodegener (2013) 0.80
Prospects for neuroprotective therapies in prodromal Huntington's disease. Mov Disord (2014) 0.80
Effects on murine behavior and lifespan of selectively decreasing expression of mutant huntingtin allele by supt4h knockdown. PLoS Genet (2015) 0.80
Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity. Hum Mol Genet (2014) 0.80
Cell-based therapies for Huntington's disease. Drug Discov Today (2014) 0.80
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The Emerging Role of Spinal Dynorphin in Chronic Pain: A Therapeutic Perspective. Annu Rev Pharmacol Toxicol (2016) 0.80
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia. Hum Mol Genet (2015) 0.80
Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice. Mol Neurodegener (2016) 0.79
Systemic delivery of P42 peptide: a new weapon to fight Huntington's disease. Acta Neuropathol Commun (2014) 0.79
Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration. Invest Ophthalmol Vis Sci (2015) 0.78
Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA. Mol Ther Nucleic Acids (2015) 0.78
Assessment of motor function, sensory motor gating and recognition memory in a novel BACHD transgenic rat model for huntington disease. PLoS One (2013) 0.78
Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain. Mol Ther Nucleic Acids (2016) 0.77
Gene therapy for misfolding protein diseases of the central nervous system. Neurotherapeutics (2013) 0.77
Antisense-mediated angiotensinogen inhibition slows polycystic kidney disease in mice with a targeted mutation in Pkd2. Am J Physiol Renal Physiol (2014) 0.77
Huntingtin is critical both pre- and postsynaptically for long-term learning-related synaptic plasticity in Aplysia. PLoS One (2014) 0.77
Identification of sequences common to more than one therapeutic target to treat complex diseases: simulating the high variance in sequence interactivity evolved to modulate robust phenotypes. BMC Genomics (2015) 0.77
scyllo-Inositol promotes robust mutant Huntingtin protein degradation. J Biol Chem (2013) 0.76
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