Published in Proc Natl Acad Sci U S A on April 08, 2008
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Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron (2012) 4.48
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A construct with fluorescent indicators for conditional expression of miRNA. BMC Biotechnol (2008) 1.08
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Design of small molecule-responsive microRNAs based on structural requirements for Drosha processing. Nucleic Acids Res (2010) 1.04
Opposing role for Egr3 in nucleus accumbens cell subtypes in cocaine action. J Neurosci (2015) 1.02
Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Genet (2013) 1.02
Neuronal dark matter: the emerging role of microRNAs in neurodegeneration. Front Cell Neurosci (2013) 1.02
MicroRNA implications across neurodevelopment and neuropathology. J Biomed Biotechnol (2009) 1.01
The dose can make the poison: lessons learned from adverse in vivo toxicities caused by RNAi overexpression. Silence (2011) 1.00
RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy. Mol Ther (2012) 1.00
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Gene therapy in animal models of autosomal dominant retinitis pigmentosa. Mol Vis (2012) 0.99
An artificial miRNA against HPSE suppresses melanoma invasion properties, correlating with a down-regulation of chemokines and MAPK phosphorylation. PLoS One (2012) 0.99
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Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype. PLoS One (2009) 0.98
A screen for enhancers of clearance identifies huntingtin as a heat shock protein 90 (Hsp90) client protein. J Biol Chem (2011) 0.98
Designing Ago2-specific siRNA/shRNA to Avoid Competition with Endogenous miRNAs. Mol Ther Nucleic Acids (2014) 0.97
Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics. Hum Mol Genet (2009) 0.97
AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy. Mol Ther (2011) 0.97
Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. Mol Ther (2013) 0.96
Targeted delivery of mutant tolerant anti-coxsackievirus artificial microRNAs using folate conjugated bacteriophage Phi29 pRNA. PLoS One (2011) 0.96
In vivo knock-down of multidrug resistance transporters ABCC1 and ABCC2 by AAV-delivered shRNAs and by artificial miRNAs. J RNAi Gene Silencing (2011) 0.96
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
Apolipoprotein B knockdown by AAV-delivered shRNA lowers plasma cholesterol in mice. Mol Ther (2011) 0.96
Regulation of the mammalian nervous system by microRNAs. Mol Pharmacol (2008) 0.96
Heritable and lineage-specific gene knockdown in zebrafish embryo. PLoS One (2009) 0.95
RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1). Mol Ther (2011) 0.94
miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther (2015) 0.94
Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy. Mol Ther (2013) 0.94
Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice. Mol Ther (2013) 0.94
RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1. Neurobiol Dis (2013) 0.94
Preclinical in vivo evaluation of the safety of a multi-shRNA-based gene therapy against HIV-1. Mol Ther Nucleic Acids (2013) 0.93
siSPOTR: a tool for designing highly specific and potent siRNAs for human and mouse. Nucleic Acids Res (2012) 0.93
The therapeutic potential of microRNAs in nervous system damage, degeneration, and repair. Neuromolecular Med (2009) 0.92
Acat1 knockdown gene therapy decreases amyloid-β in a mouse model of Alzheimer's disease. Mol Ther (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
Knockdown of orexin type 1 receptor in rat locus coeruleus increases REM sleep during the dark period. Eur J Neurosci (2010) 0.91
An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases. BMC Mol Biol (2012) 0.91
Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease. Mol Ther (2013) 0.91
Moderate long-term modulation of neuropeptide Y in hypothalamic arcuate nucleus induces energy balance alterations in adult rats. PLoS One (2011) 0.91
Establishment and characterization of Prnp knockdown neuroblastoma cells using dual microRNA-mediated RNA interference. Prion (2011) 0.91
Oligonucleotide-based strategies to combat polyglutamine diseases. Nucleic Acids Res (2014) 0.90
Silencing of Parkinson's disease-associated genes with artificial mirtron mimics of miR-1224. Nucleic Acids Res (2012) 0.90
siRNA-based therapeutic approaches for rheumatic diseases. Nat Rev Rheumatol (2012) 0.90
"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease. Neuron (2012) 0.90
Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model. Mol Ther (2012) 0.89
Striatal CB1 and D2 receptors regulate expression of each other, CRIP1A and δ opioid systems. J Neurochem (2013) 0.89
Improved siRNA/shRNA functionality by mismatched duplex. PLoS One (2011) 0.89
Quantification assays for total and polyglutamine-expanded huntingtin proteins. PLoS One (2014) 0.88
Lentiviral miR30-based RNA interference against heparanase suppresses melanoma metastasis with lower liver and lung toxicity. Int J Biol Sci (2013) 0.88
Advances with microRNAs in Parkinson's disease research. Drug Des Devel Ther (2013) 0.87
Constitutive expression of short hairpin RNA in vivo triggers buildup of mature hairpin molecules. Hum Gene Ther (2011) 0.87
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther (2015) 0.87
Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy. Mol Ther (2012) 0.86
Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6. Neurobiol Dis (2011) 0.86
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An abundant class of tiny RNAs with probable regulatory roles in Caenorhabditis elegans. Science (2001) 44.16
The 21-nucleotide let-7 RNA regulates developmental timing in Caenorhabditis elegans. Nature (2000) 39.31
An extensive class of small RNAs in Caenorhabditis elegans. Science (2001) 34.17
Functional siRNAs and miRNAs exhibit strand bias. Cell (2003) 19.15
Rational siRNA design for RNA interference. Nat Biotechnol (2004) 14.16
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature (2006) 10.95
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RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A (2005) 4.79
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med (2004) 4.63
The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease. J Neurosci (2008) 3.62
Allele-specific silencing of dominant disease genes. Proc Natl Acad Sci U S A (2003) 3.00
Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther (2004) 2.84
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Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther (2009) 2.47
Identification of PDGFR as a receptor for AAV-5 transduction. Nat Med (2003) 2.43
MicroRNAs potentiate neural development. Neuron (2009) 2.25
Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation (2003) 2.22
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther (2005) 2.08
Restoring Acid-sensing ion channel-1a in the amygdala of knock-out mice rescues fear memory but not unconditioned fear responses. J Neurosci (2008) 1.90
DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo. Ann Neurol (2010) 1.89
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Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis. Mol Ther (2008) 1.82
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Targeted viral delivery of Cre recombinase induces conditional gene deletion in cardiovascular circuits of the mouse brain. Physiol Genomics (2004) 1.74
Functional repair of human donor lungs by IL-10 gene therapy. Sci Transl Med (2009) 1.72
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther (2011) 1.72
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Silencing primary dystonia: lentiviral-mediated RNA interference therapy for DYT1 dystonia. J Neurosci (2005) 1.53
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A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther (2002) 1.40
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Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant alleles. Nucleic Acids Res (2004) 1.27
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Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice. Mol Ther (2005) 1.10
Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol Ther (2011) 1.09
RNAi-based therapeutics targeting survivin and PLK1 for treatment of bladder cancer. Mol Ther (2011) 1.09
Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimer's disease. Mol Ther (2009) 1.09
Intraarticular gene transfer of TNFR:Fc suppresses experimental arthritis with reduced systemic distribution of the gene product. Mol Ther (2002) 1.05
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Gene transfer to the nonhuman primate retina with recombinant feline immunodeficiency virus vectors. Hum Gene Ther (2002) 1.04
What does it take to bind CAR? Mol Ther (2005) 1.03
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RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy. Mol Ther (2012) 1.00
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Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinA. Ann Neurol (2003) 1.00
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Evolution of alternative splicing in primate brain transcriptomes. Hum Mol Genet (2010) 0.99
Large-scale analysis of exonized mammalian-wide interspersed repeats in primate genomes. Hum Mol Genet (2009) 0.99
In vivo SELEX for Identification of Brain-penetrating Aptamers. Mol Ther Nucleic Acids (2013) 0.98
RNAi therapeutics for CNS disorders. Brain Res (2010) 0.97
Generation of hairpin-based RNAi vectors for biological and therapeutic application. Methods Enzymol (2012) 0.97
Viral-mediated gene transfer to mouse primary neural progenitor cells. Mol Ther (2002) 0.97
An amino acid-based amphoteric liposomal delivery system for systemic administration of siRNA. Mol Ther (2011) 0.97
PP2A:B56{epsilon}, a substrate of caspase-3, regulates p53-dependent and p53-independent apoptosis during development. J Biol Chem (2010) 0.96
RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1). Mol Ther (2011) 0.94