Antisense oligonucleotides: treating neurodegeneration at the level of RNA

Conserved Seed Pairing, Often Flanked by Adenosines, Indicates that Thousands of Human Genes are MicroRNA Targets

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Amyloid oligomers exacerbate tau pathology in a mouse model of tauopathy

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Alternative isoform regulation in human tissue transcriptomes

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The HCV Life Cycle: In vitro Tissue Culture Systems and Therapeutic Targets

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The widespread regulation of microRNA biogenesis, function and decay

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Silencing of microRNAs in vivo with ‘antagomirs’

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Gene silencing by microRNAs: contributions of translational repression and mRNA decay

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Treatment of HCV Infection by Targeting MicroRNA

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Alzheimer Disease in the US Population: Prevalence Estimates Using the 2000 Census

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miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting

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Reducing Endogenous Tau Ameliorates Amyloid ß-Induced Deficits in an Alzheimer's Disease Mouse Model

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Dendritic Function of Tau Mediates Amyloid-β Toxicity in Alzheimer's Disease Mouse Models

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Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection

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LNA-mediated microRNA silencing in non-human primates

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A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

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RNA and Disease

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LNA (Locked Nucleic Acids): Synthesis of the adenine, cytosine, guanine, 5-methylcytosine, thymine and uracil bicyclonucleoside monomers, oligomerisation, and unprecedented nucleic acid recognition

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Cloning and sequencing of the cDNA encoding an isoform of microtubule‐associated protein tau containing four tandem repeats: differential expression of tau protein mRNAs in human brain.

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MicroRNA-143 Regulates Adipocyte Differentiation

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Antisense technologies

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Mutation-Specific Functional Impairments in Distinct Tau Isoforms of Hereditary FTDP-17

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Expression of separate isoforms of human tau protein: correlation with the tau pattern in brain and effects on tubulin polymerization.

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Small Interfering RNA-Induced Transcriptional Gene Silencing in Human Cells

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Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes

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Ribonuclease H: the enzymes in eukaryotes

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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

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

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Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

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Transport of drugs across the blood–brain barrier by nanoparticles.

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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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Inhibition of natural antisense transcripts in vivo results in gene-specific transcriptional upregulation

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Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelPreprint

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Morpholino antisense oligomers: the case for an RNase H-independent structural type

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Dystrophin Immunity in Duchenne's Muscular Dystrophy

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Amyloid-β/Fyn–Induced Synaptic, Network, and Cognitive Impairments Depend on Tau Levels in Multiple Mouse Models of Alzheimer's Disease

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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

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Structure and novel exons of the human .tau. gene

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

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Developmentally regulated expression of specific tau sequences

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Tau deficiency induces parkinsonism with dementia by impairing APP-mediated iron export

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Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice

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Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

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

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Missense and silent tau gene mutations cause frontotemporal dementia with parkinsonism-chromosome 17 type, by affecting multiple alternative RNA splicing regulatory elements.

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Inhibition of neuronal maturation in primary hippocampal neurons from tau deficient mice

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Neurotrophins are required for nerve growth during development

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Activating gene expression in mammalian cells with promoter-targeted duplex RNAs

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Antisense oligonucleotide therapy for neurodegenerative disease

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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

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Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals

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Efficient Reduction of Target RNAs by Small Interfering RNA and RNase H-dependent Antisense Agents

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The Centrality of RNA

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Uniformly modified 2'-deoxy-2'-fluoro-phosphorothioate oligonucleotides as nuclease-resistant antisense compounds with high affinity and specificity for RNA targets

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ANTISENSE OLIGONUCLEOTIDES: FROM DESIGN TO THERAPEUTIC APPLICATION

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Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.

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Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

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Efficacy of systemic morpholino exon‐skipping in duchenne dystrophy dogs

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Phosphorothioate Oligodeoxynucleotides: What Is Their Origin and What Is Unique About Them?

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Comparative Biochemistry of Tau in Progressive Supranuclear Palsy, Corticobasal Degeneration, FTDP‐17 and Pick's Disease

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Determination of the Role of the Human RNase H1 in the Pharmacology of DNA-like Antisense Drugs

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microRNA‐34c is a novel target to treat dementias

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Risks and benefits of thoracic epidural anaesthesia

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Apolipoprotein E4 Causes Age- and Tau-Dependent Impairment of GABAergic Interneurons, Leading to Learning and Memory Deficits in Mice

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miR‐206 regulates brain‐derived neurotrophic factor in Alzheimer disease model

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Systemically delivered antisense oligomers upregulate gene expression in mouse tissues

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

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Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

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Inhibition of endothelial cell adhesion molecule expression with antisense oligonucleotides.

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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

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Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

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

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Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

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Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

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Mechanisms of Antisense Transcription Initiation with Implications in Gene Expression, Genomic Integrity and Disease Pathogenesis

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Antisense Oligonucleotide in LNA-Gapmer Design Targeting TGFBR2—A Key Single Gene Target for Safe and Effective Inhibition of TGFβ Signaling

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Modified Gold Nanoparticles to Overcome the Chemoresistance to Gemcitabine in Mutant p53 Cancer Cells

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Tau Protein as Therapeutic Target for Cancer? Focus on Glioblastoma

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Novel Therapeutic Horizons: SNCA Targeting in Parkinson’s Disease

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An Antisense Oligonucleotide Leads to Suppressed Transcription of Hdac2 and Long-Term Memory Enhancement

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An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy

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Metallacarborane Complex Boosts the Rate of DNA Oligonucleotide Hydrolysis in the Reaction Catalyzed by Snake Venom Phosphodiesterase

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FTLD Treatment: Current Practice and Future Possibilities

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