Published in J Med Chem on April 20, 2006
Inhibiting gene expression with peptide nucleic acid (PNA)--peptide conjugates that target chromosomal DNA. Biochemistry (2007) 1.33
Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide. Nucleic Acids Res (2007) 1.28
Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA. Mol Ther Nucleic Acids (2012) 0.95
Peptide nucleic acids conjugated to short basic peptides show improved pharmacokinetics and antisense activity in adipose tissue. J Med Chem (2010) 0.86
A versatile method for the preparation of conjugates of peptides with DNA/PNA/analog by employing chemo-selective click reaction in water. Nucleic Acids Res (2007) 0.85
Immunological response to peptide nucleic acid and its peptide conjugate targeted to transactivation response (TAR) region of HIV-1 RNA genome. Oligonucleotides (2008) 0.81
Silencing of microRNAs in vivo with 'antagomirs'. Nature (2005) 32.56
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature (2004) 12.75
miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting. Cell Metab (2006) 12.41
miR-181a is an intrinsic modulator of T cell sensitivity and selection. Cell (2007) 9.08
The nuclear-retained noncoding RNA MALAT1 regulates alternative splicing by modulating SR splicing factor phosphorylation. Mol Cell (2010) 8.66
RNAi-mediated gene silencing in non-human primates. Nature (2006) 7.60
Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7. Nat Med (2005) 7.18
A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol (2008) 6.81
MicroRNA-143 regulates adipocyte differentiation. J Biol Chem (2004) 6.78
Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43. Nat Neurosci (2011) 6.01
Regulating gene expression through RNA nuclear retention. Cell (2005) 5.58
MicroRNA 21 promotes glioma invasion by targeting matrix metalloproteinase regulators. Mol Cell Biol (2008) 5.21
A viral microRNA functions as an orthologue of cellular miR-155. Nature (2007) 5.09
Rational design of cationic lipids for siRNA delivery. Nat Biotechnol (2010) 4.97
RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol (2010) 4.86
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc Natl Acad Sci U S A (2008) 4.57
Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron (2012) 4.48
RNAi therapeutics: a potential new class of pharmaceutical drugs. Nat Chem Biol (2006) 4.38
Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides. Nature (2011) 4.34
Antagonism of miR-33 in mice promotes reverse cholesterol transport and regression of atherosclerosis. J Clin Invest (2011) 4.21
Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model. Nature (2011) 4.04
Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat Biotechnol (2007) 4.02
Specificity, duplex degradation and subcellular localization of antagomirs. Nucleic Acids Res (2007) 3.89
Integration of microRNA miR-122 in hepatic circadian gene expression. Genes Dev (2009) 3.64
Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. A comparative analysis. J Biol Chem (2002) 3.32
Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol Ther (2010) 3.26
RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention. Neuron (2013) 3.25
Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am J Hum Genet (2008) 3.24
Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery. Nat Nanotechnol (2012) 3.09
Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev (2010) 3.02
Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Invest (2006) 3.00
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
Single-stranded siRNAs activate RNAi in animals. Cell (2012) 2.64
Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature (2012) 2.63
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci Transl Med (2011) 2.56
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
Development of lipidoid-siRNA formulations for systemic delivery to the liver. Mol Ther (2009) 2.39
Systemically delivered antisense oligomers upregulate gene expression in mouse tissues. Nat Biotechnol (2002) 2.38
A bioinformatics based approach to discover small RNA genes in the Escherichia coli genome. Biosystems (2002) 2.31
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon. PLoS Biol (2007) 2.29
Prelamin A and lamin A appear to be dispensable in the nuclear lamina. J Clin Invest (2006) 2.23
Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA. J Med Chem (2005) 2.05
Potent inhibition of microRNA in vivo without degradation. Nucleic Acids Res (2008) 1.98
Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals. Nucleic Acids Res (2006) 1.98
Durable protection from Herpes Simplex Virus-2 transmission following intravaginal application of siRNAs targeting both a viral and host gene. Cell Host Microbe (2009) 1.90
Rapid identification of emerging pathogens: coronavirus. Emerg Infect Dis (2005) 1.87
Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo. Angew Chem Int Ed Engl (2012) 1.87
Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes. Nucleic Acids Res (2011) 1.84
Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin. Mol Ther (2011) 1.82
MyomiR-133 regulates brown fat differentiation through Prdm16. Nat Cell Biol (2012) 1.80
Influenza A virus-generated small RNAs regulate the switch from transcription to replication. Proc Natl Acad Sci U S A (2010) 1.77
Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway. Nature (2007) 1.66
Intravesical delivery of small activating RNA formulated into lipid nanoparticles inhibits orthotopic bladder tumor growth. Cancer Res (2012) 1.65
Short antisense oligonucleotides with novel 2'-4' conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals. J Med Chem (2009) 1.61
RNA interference-mediated silencing of the respiratory syncytial virus nucleocapsid defines a potent antiviral strategy. Antimicrob Agents Chemother (2009) 1.60
SAR by MS: discovery of a new class of RNA-binding small molecules for the hepatitis C virus: internal ribosome entry site IIA subdomain. J Med Chem (2005) 1.59
Positional effect of chemical modifications on short interference RNA activity in mammalian cells. J Med Chem (2005) 1.57
Structure-activity relationship of heterobase-modified 2'-C-methyl ribonucleosides as inhibitors of hepatitis C virus RNA replication. J Med Chem (2004) 1.57
Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2'-O-(2-methoxyethyl) modified antisense oligonucleotide: comparison across species. Drug Metab Dispos (2003) 1.57
Antisense oligonucleotide-based therapeutics for cancer. Oncogene (2003) 1.54
Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat. Biochemistry (2010) 1.50
Competition for RISC binding predicts in vitro potency of siRNA. Nucleic Acids Res (2006) 1.44
Misregulated RNA processing in amyotrophic lateral sclerosis. Brain Res (2012) 1.42
Structure-activity relationship of purine ribonucleosides for inhibition of hepatitis C virus RNA-dependent RNA polymerase. J Med Chem (2004) 1.41
RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1. Proc Natl Acad Sci U S A (2012) 1.40
Synthesis and biophysical evaluation of 2',4'-constrained 2'O-methoxyethyl and 2',4'-constrained 2'O-ethyl nucleic acid analogues. J Org Chem (2010) 1.36
hsa-miR-191 is a candidate oncogene target for hepatocellular carcinoma therapy. Cancer Res (2010) 1.35
Gene silencing activity of siRNAs with a ribo-difluorotoluyl nucleotide. ACS Chem Biol (2006) 1.33
Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J Am Chem Soc (2014) 1.31
Antisense reduction of tau in adult mice protects against seizures. J Neurosci (2013) 1.29
Unexpected origins of the enhanced pairing affinity of 2'-fluoro-modified RNA. Nucleic Acids Res (2010) 1.28
Therapeutic silencing of microRNA-33 inhibits the progression of atherosclerosis in Ldlr-/- mice--brief report. Arterioscler Thromb Vasc Biol (2013) 1.28
Antisense-based therapy for the treatment of spinal muscular atrophy. J Cell Biol (2012) 1.25
Promoter RNA links transcriptional regulation of inflammatory pathway genes. Nucleic Acids Res (2013) 1.24
Systemic RNAi-mediated Gene Silencing in Nonhuman Primate and Rodent Myeloid Cells. Mol Ther Nucleic Acids (2012) 1.24
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
Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing. Nat Chem Biol (2012) 1.22
An SREBP-responsive microRNA operon contributes to a regulatory loop for intracellular lipid homeostasis. Cell Metab (2013) 1.20
Activation of LDL receptor expression by small RNAs complementary to a noncoding transcript that overlaps the LDLR promoter. Chem Biol (2010) 1.20
Human RNase H1 discriminates between subtle variations in the structure of the heteroduplex substrate. Mol Pharmacol (2006) 1.18
Modulation of thermal stability can enhance the potency of siRNA. Nucleic Acids Res (2010) 1.14
Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases. Trends Mol Med (2012) 1.14
Crystal structure, stability and in vitro RNAi activity of oligoribonucleotides containing the ribo-difluorotoluyl nucleotide: insights into substrate requirements by the human RISC Ago2 enzyme. Nucleic Acids Res (2007) 1.11