Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.

PubWeight™: 3.07‹?› | Rank: Top 1%

🔗 View Article (PMC 2040405)

Published in Proc Natl Acad Sci U S A on October 16, 2007

Authors

M DiFiglia1, M Sena-Esteves, K Chase, E Sapp, E Pfister, M Sass, J Yoder, P Reeves, R K Pandey, K G Rajeev, M Manoharan, D W Y Sah, P D Zamore, N Aronin

Author Affiliations

1: Department of Neurology, Massachusetts General Hospital, Charlestown, MA 02114, USA.

Articles citing this

(truncated to the top 100)

Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov (2009) 12.46

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron (2012) 4.48

Mitochondria in neuroplasticity and neurological disorders. Neuron (2008) 3.35

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell (2012) 2.83

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. Nat Biotechnol (2009) 2.36

Prion-like disorders: blurring the divide between transmissibility and infectivity. J Cell Sci (2010) 2.29

Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients. Curr Biol (2009) 2.19

A status report on RNAi therapeutics. Silence (2010) 2.18

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus. Proc Natl Acad Sci U S A (2010) 1.92

Mesenchymal stem cells for the treatment of neurodegenerative disease. Regen Med (2010) 1.89

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

CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup. Am J Hum Genet (2009) 1.78

Identification of NUB1 as a suppressor of mutant Huntington toxicity via enhanced protein clearance. Nat Neurosci (2013) 1.71

Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease. Nat Med (2014) 1.67

Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol Ther (2009) 1.66

Systemic exosomal siRNA delivery reduced alpha-synuclein aggregates in brains of transgenic mice. Mov Disord (2014) 1.63

Progress toward in vivo use of siRNAs-II. Mol Ther (2011) 1.60

Oligonucleotide therapeutic approaches for Huntington disease. J Clin Invest (2011) 1.54

Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein. Nat Biotechnol (2010) 1.38

Mitochondrial structural and functional dynamics in Huntington's disease. Brain Res Rev (2009) 1.37

Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism. Chem Biol (2010) 1.36

Allele-specific silencing of mutant Huntington's disease gene. J Neurochem (2009) 1.35

Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease. PLoS One (2008) 1.34

Lipidic systems for in vivo siRNA delivery. AAPS J (2009) 1.33

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease. Mol Ther (2008) 1.31

Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats. Nucleic Acids Res (2011) 1.27

Nanoparticle-based delivery of small interfering RNA: challenges for cancer therapy. Int J Nanomedicine (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

Targets for future clinical trials in Huntington's disease: what's in the pipeline? Mov Disord (2014) 1.21

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

Linking SNPs to CAG repeat length in Huntington's disease patients. Nat Methods (2008) 1.19

In vivo silencing of alpha-synuclein using naked siRNA. Mol Neurodegener (2008) 1.18

Mutant huntingtin, abnormal mitochondrial dynamics, defective axonal transport of mitochondria, and selective synaptic degeneration in Huntington's disease. Biochim Biophys Acta (2011) 1.16

siRNA knock-down of mutant torsinA restores processing through secretory pathway in DYT1 dystonia cells. Hum Mol Genet (2008) 1.15

Behavioral epigenetics. Ann N Y Acad Sci (2011) 1.14

HTT-lowering reverses Huntington's disease immune dysfunction caused by NFκB pathway dysregulation. Brain (2014) 1.13

Genetically engineered mesenchymal stem cells as a proposed therapeutic for Huntington's disease. Mol Neurobiol (2011) 1.11

Huntington's Disease. Cold Spring Harb Perspect Biol (2011) 1.11

RNAi: a potential new class of therapeutic for human genetic disease. Hum Genet (2011) 1.10

Polymers in small-interfering RNA delivery. Nucleic Acid Ther (2011) 1.09

Allele-selective inhibition of trinucleotide repeat genes. Drug Discov Today (2012) 1.08

Delivery of small interfering RNA by peptide-targeted mesoporous silica nanoparticle-supported lipid bilayers. ACS Nano (2012) 1.08

Full-length huntingtin levels modulate body weight by influencing insulin-like growth factor 1 expression. Hum Mol Genet (2010) 1.06

Huntington's disease: progress toward effective disease-modifying treatments and a cure. Hum Mol Genet (2010) 1.05

Tiagabine is neuroprotective in the N171-82Q and R6/2 mouse models of Huntington's disease. Neurobiol Dis (2008) 1.05

Emergence and natural selection of drug-resistant prions. Mol Biosyst (2010) 1.05

Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA. Ann N Y Acad Sci (2009) 1.05

A fully humanized transgenic mouse model of Huntington disease. Hum Mol Genet (2012) 1.01

Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins. Prog Neurobiol (2011) 1.00

The role of ALFY in selective autophagy. Cell Death Differ (2012) 1.00

RNAi medicine for the brain: progresses and challenges. Hum Mol Genet (2011) 1.00

Preclinical development of siRNA therapeutics for AL amyloidosis. Gene Ther (2011) 0.99

Huntington's disease and the striatal medium spiny neuron: cell-autonomous and non-cell-autonomous mechanisms of disease. Neurotherapeutics (2012) 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

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides. Mol Ther (2014) 0.97

Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics. Hum Mol Genet (2009) 0.97

Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice. PLoS One (2013) 0.96

Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression. J Biol Chem (2008) 0.96

Huntington's disease and its therapeutic target genes: a global functional profile based on the HD Research Crossroads database. BMC Neurol (2012) 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

Early or late-stage anti-N-terminal Huntingtin intrabody gene therapy reduces pathological features in B6.HDR6/1 mice. J Neuropathol Exp Neurol (2010) 0.95

Silencing human genetic diseases with oligonucleotide-based therapies. Hum Genet (2013) 0.95

High-content chemical and RNAi screens for suppressors of neurotoxicity in a Huntington's disease model. PLoS One (2011) 0.95

Focused ultrasound for targeted delivery of siRNA and efficient knockdown of Htt expression. J Control Release (2012) 0.94

RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression. Nucleic Acids Res (2013) 0.94

Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin. Mol Cell Neurosci (2011) 0.94

Allele-selective inhibition of expression of huntingtin and ataxin-3 by RNA duplexes containing unlocked nucleic acid substitutions. Biochemistry (2013) 0.93

Longitudinal behavioral, cross-sectional transcriptional and histopathological characterization of a knock-in mouse model of Huntington's disease with 140 CAG repeats. Exp Neurol (2010) 0.93

Integration of β-catenin, sirtuin, and FOXO signaling protects from mutant huntingtin toxicity. J Neurosci (2012) 0.92

Neuroprotection for Huntington's disease: ready, set, slow. Neurotherapeutics (2008) 0.92

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Mol Ther Nucleic Acids (2015) 0.91

An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases. BMC Mol 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

High-density lipoprotein facilitates in vivo delivery of α-tocopherol-conjugated short-interfering RNA to the brain. Hum Gene Ther (2011) 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

Antisense therapeutics in oncology: current status. Onco Targets Ther (2014) 0.89

Huntington's disease: silencing a brutal killer. Exp Neurol (2009) 0.89

Emerging role of non-coding RNA in neural plasticity, cognitive function, and neuropsychiatric disorders. Front Genet (2012) 0.89

Quantification assays for total and polyglutamine-expanded huntingtin proteins. PLoS One (2014) 0.88

Antisense therapy in neurology. J Pers Med (2013) 0.87

Huntingtin is required for normal excitatory synapse development in cortical and striatal circuits. J Neurosci (2014) 0.87

Intrabodies as neuroprotective therapeutics. Neurotherapeutics (2013) 0.87

Transgenic animal models for study of the pathogenesis of Huntington's disease and therapy. Drug Des Devel Ther (2015) 0.86

Huntington's disease: the past, present, and future search for disease modifiers. Yale J Biol Med (2013) 0.85

Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels. J Clin Invest (2016) 0.85

Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors. Mol Neurodegener (2010) 0.85

Use of genetically modified mesenchymal stem cells to treat neurodegenerative diseases. Int J Mol Sci (2014) 0.84

Therapeutic approaches to preventing cell death in Huntington disease. Prog Neurobiol (2012) 0.84

Experimental animal model and RNA interference: a promising association for bladder cancer research. World J Urol (2009) 0.84

Epigenetic mechanisms of neurodegeneration in Huntington's disease. Neurotherapeutics (2013) 0.83

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

High-density lipoproteins for the systemic delivery of short interfering RNA. Expert Opin Drug Deliv (2013) 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

Mass spectrometric identification of novel lysine acetylation sites in huntingtin. Mol Cell Proteomics (2011) 0.81

Gene therapy in mouse models of huntington disease. Neuroscientist (2011) 0.81

Mutant huntingtin gene-dose impacts on aggregate deposition, DARPP32 expression and neuroinflammation in HdhQ150 mice. PLoS One (2013) 0.81

Lipoproteins and lipoprotein mimetics for imaging and drug delivery. Adv Drug Deliv Rev (2016) 0.81

GluN3A: an NMDA receptor subunit with exquisite properties and functions. Neural Plast (2013) 0.81

Articles cited by this

Silencing of microRNAs in vivo with 'antagomirs'. Nature (2005) 32.56

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group. Cell (1993) 28.99

Asymmetry in the assembly of the RNAi enzyme complex. Cell (2003) 22.18

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature (2004) 12.75

Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death. Nature (2004) 11.82

Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain. Science (1997) 11.53

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature (2006) 10.95

Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation. Cell (1997) 9.57

Huntingtin acts in the nucleus to induce apoptosis but death does not correlate with the formation of intranuclear inclusions. Cell (1998) 7.13

Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease. Cell (2000) 6.45

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol (2005) 6.40

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

Nuclear and neuropil aggregates in Huntington's disease: relationship to neuropathology. J Neurosci (1999) 4.27

Neurological abnormalities in a knock-in mouse model of Huntington's disease. Hum Mol Genet (2001) 3.84

Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons. Neuron (1995) 3.08

Allele-specific silencing of dominant disease genes. Proc Natl Acad Sci U S A (2003) 3.00

Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med (2005) 2.80

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med (2005) 2.80

Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat Neurosci (2005) 2.75

Using siRNA in prophylactic and therapeutic regimens against SARS coronavirus in Rhesus macaque. Nat Med (2005) 2.55

Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther (2005) 2.47

Comparison of siRNA-induced off-target RNA and protein effects. RNA (2007) 2.22

Induction of the interferon response by siRNA is cell type- and duplex length-dependent. RNA (2006) 2.17

siRNA relieves chronic neuropathic pain. Nucleic Acids Res (2004) 1.96

Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis. Aging Cell (2003) 1.88

Lack of interferon response in animals to naked siRNAs. Nat Biotechnol (2004) 1.71

Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci Res (2005) 1.68

Neurochemical and behavioral consequences of widespread gene knockdown in the adult mouse brain by using nonviral RNA interference. Proc Natl Acad Sci U S A (2004) 1.58

Changes in cortical and striatal neurons predict behavioral and electrophysiological abnormalities in a transgenic murine model of Huntington's disease. J Neurosci (2001) 1.52

rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse. Biochem Biophys Res Commun (2006) 1.45

Adeno-associated virus (AAV) vectors in the CNS. Curr Gene Ther (2005) 1.43

Mutant huntingtin expression in clonal striatal cells: dissociation of inclusion formation and neuronal survival by caspase inhibition. J Neurosci (1999) 1.40

Knock-in mouse models of Huntington's disease. NeuroRx (2005) 1.40

Lentiviral-mediated delivery of mutant huntingtin in the striatum of rats induces a selective neuropathology modulated by polyglutamine repeat size, huntingtin expression levels, and protein length. J Neurosci (2002) 1.40

Stereology: a practical primer for neuropathology. J Neuropathol Exp Neurol (1998) 1.37

Progressive loss of BDNF in a mouse model of Huntington's disease and rescue by BDNF delivery. Pharmacol Res (2005) 1.35

mRNA for low density lipoprotein receptor in brain and spinal cord of immature and mature rabbits. Proc Natl Acad Sci U S A (1987) 1.14

Expressing short hairpin RNAs in vivo. Nat Methods (2006) 1.14

Delivery of siRNA and siRNA expression constructs to adult mammals by hydrodynamic intravascular injection. Methods Enzymol (2005) 1.10

Enhanced hepatic uptake and bioactivity of type alpha1(I) collagen gene promoter-specific triplex-forming oligonucleotides after conjugation with cholesterol. J Pharmacol Exp Ther (2006) 1.02

Articles by these authors

A cellular function for the RNA-interference enzyme Dicer in the maturation of the let-7 small temporal RNA. Science (2001) 31.33

Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain. Science (1997) 11.53

ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell (2001) 10.20

Targeted mRNA degradation by double-stranded RNA in vitro. Genes Dev (1999) 8.21

A factor, U2AF, is required for U2 snRNP binding and splicing complex assembly. Cell (1988) 6.16

Genetics of resistance to colicins in Escherichia coli K-12: cross-resistance among colicins of group A. J Bacteriol (1975) 5.67

Outer membrane proteins of Escherichia coli. VI. Protein alteration in bacteriophage-resistant mutants. J Bacteriol (1977) 5.16

Genetics of resistance to colicins in Escherichia coli K-12: cross-resistance among colicins of group B. J Bacteriol (1975) 4.94

Molecular biology. RNA interference. Science (2000) 4.87

Con--mutants: class of mutants in Escherichia coli K-12 lacking a major cell wall protein and defective in conjugation and adsorption of a bacteriophage. J Bacteriol (1974) 4.63

Case definitions, diagnostic algorithms, and priorities in encephalitis: consensus statement of the international encephalitis consortium. Clin Infect Dis (2013) 4.51

The protein Sex-lethal antagonizes the splicing factor U2AF to regulate alternative splicing of transformer pre-mRNA. Nature (1993) 3.97

Structure of the O antigen of Escherichia coli K-12 and the sequence of its rfb gene cluster. J Bacteriol (1994) 3.61

The tolC locus of Escherichia coli affects the expression of three major outer membrane proteins. J Bacteriol (1982) 3.51

Mammalian nuclei contain foci which are highly enriched in components of the pre-mRNA splicing machinery. EMBO J (1991) 3.49

Characterization of group B colicin-resistant mutants of Escherichia coli K-12: colicin resistance and the role of enterochelin. J Bacteriol (1976) 3.19

Huntingtin expression stimulates endosomal-lysosomal activity, endosome tubulation, and autophagy. J Neurosci (2000) 3.16

Translational regulation in development. Cell (1995) 3.13

Genetic locus (ompB) affecting a major outer-membrane protein in Escherichia coli K-12. J Bacteriol (1977) 3.10

Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons. Neuron (1995) 3.08

Iron uptake in colicin B-resistant mutants of Escherichia coli K-12. J Bacteriol (1976) 2.95

Bacteriophage resistance in Escherichia coli K-12: general pattern of resistance. J Bacteriol (1975) 2.94

Cloning and intracellular localization of the U2 small nuclear ribonucleoprotein auxiliary factor small subunit. Proc Natl Acad Sci U S A (1992) 2.82

Mutants resistant to colicin CA42-E2: cross resistance and genetic mapping of a special class of mutants. Aust J Exp Biol Med Sci (1966) 2.38

Crystal structure of a Pumilio homology domain. Mol Cell (2001) 2.32

Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain. Neuroscience (2006) 2.19

Early and progressive accumulation of reactive microglia in the Huntington disease brain. J Neuropathol Exp Neurol (2001) 2.17

Interactions between quantitative trait loci in soybean in which trait variation at one locus is conditional upon a specific allele at another. Proc Natl Acad Sci U S A (1995) 2.08

Uptake of ferrienterochelin by Escherichia coli: energy dependent stage of uptake. J Bacteriol (1977) 2.07

Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Hum Mol Genet (1998) 2.07

Dissected nucleus-independent chemical shift analysis of pi-aromaticity and antiaromaticity. Org Lett (2001) 2.01

Environmental iodine intake and thyroid dysfunction during chronic amiodarone therapy. Ann Intern Med (1984) 2.01

Identification and characterization of the TolC protein, an outer membrane protein from Escherichia coli. J Bacteriol (1983) 2.00

Caspase 3-cleaved N-terminal fragments of wild-type and mutant huntingtin are present in normal and Huntington's disease brains, associate with membranes, and undergo calpain-dependent proteolysis. Proc Natl Acad Sci U S A (2001) 1.99

Triosephosphate isomerase catalysis is diffusion controlled. Appendix: Analysis of triose phosphate equilibria in aqueous solution by 31P NMR. Biochemistry (1988) 1.97

The role of colicin receptors in the uptake of ferrienterochelin by Escherichia coli K-12. Biochem Biophys Res Commun (1977) 1.96

Outer membrane of Escherichia coli K-12: isolation of mutants with altered protein 3A by using host range mutants of bacteriophage K3. J Bacteriol (1976) 1.94

Identification and sequence of rfbS and rfbE, which determine antigenic specificity of group A and group D salmonellae. J Bacteriol (1989) 1.92

Lipopolysaccharide-deficient, bacteriophage-resistant mutants of Escherichia coli K-12. J Bacteriol (1976) 1.86

Wild-type and mutant huntingtins function in vesicle trafficking in the secretory and endocytic pathways. Exp Neurol (1998) 1.85

Huntingtin localization in brains of normal and Huntington's disease patients. Ann Neurol (1997) 1.82

Defective growth functions in mutants of Escherichia coli K12 lacking a major outer membrane protein. J Mol Biol (1977) 1.75

Pharmacokinetic properties of 2'-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats. J Pharmacol Exp Ther (2001) 1.73

Identification and sequence of the gene for abequose synthase, which confers antigenic specificity on group B salmonellae: homology with galactose epimerase. J Bacteriol (1989) 1.71

Mediation of serum resistance in Salmonella typhimurium by an 11-kilodalton polypeptide encoded by the cryptic plasmid. J Infect Dis (1987) 1.70

Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs. Nucleic Acids Res (2001) 1.67

Primary structure of the tolC gene that codes for an outer membrane protein of Escherichia coli K12. Nucleic Acids Res (1983) 1.65

Crystal structure and improved antisense properties of 2'-O-(2-methoxyethyl)-RNA. Nat Struct Biol (1999) 1.65

MicroRNA therapeutics. Gene Ther (2011) 1.61

Inherited infantile dilated cardiomyopathy in dogs: genetic, clinical, biochemical, and morphologic findings. Am J Med Genet (2000) 1.61

An in vivo quantitative structure-activity relationship for a congeneric series of pyropheophorbide derivatives as photosensitizers for photodynamic therapy. Cancer Res (1997) 1.60

Gene expression profiling identifies FKBP39 as an inhibitor of autophagy in larval Drosophila fat body. Cell Death Differ (2007) 1.58

Pseudo--half-knot formation with RNA. Science (1992) 1.58

A comparative evaluation of intranasal midazolam, ketamine and their combination for sedation of young uncooperative pediatric dental patients: a triple blind randomized crossover trial. J Clin Pediatr Dent (2011) 1.56

Regulation of the pho regulon of Escherichia coli K-12. Cloning of the regulatory genes phoB and phoR and identification of their gene products. J Mol Biol (1982) 1.55

Changes in cortical and striatal neurons predict behavioral and electrophysiological abnormalities in a transgenic murine model of Huntington's disease. J Neurosci (2001) 1.52

Detection of alkali metal ions in DNA crystals using state-of-the-art X-ray diffraction experiments. Nucleic Acids Res (2001) 1.49

A CCHC metal-binding domain in Nanos is essential for translational regulation. EMBO J (1997) 1.49

Increased production of the outer membrane receptors for colicins B, D and M by Escherichia coli under iron starvation. Biochem Biophys Res Commun (1976) 1.48

Impacts of urbanisation on the lifestyle and on the prevalence of diabetes in native Asian Indian population. Diabetes Res Clin Pract (1999) 1.48

Periplasmic maltose-binding protein confers specificity on the outer membrane maltose pore of Escherichia coli. J Bacteriol (1980) 1.47

Neural precursor cells for delivery of replication-conditional HSV-1 vectors to intracerebral gliomas. Mol Ther (2000) 1.45

Functional coexpression of HSV-1 thymidine kinase and green fluorescent protein: implications for noninvasive imaging of transgene expression. Neoplasia (1999) 1.44

Distribution and elimination of Photofrin II in mice. Photochem Photobiol (1989) 1.43

EGFRvIII promotes glioma angiogenesis and growth through the NF-κB, interleukin-8 pathway. Oncogene (2011) 1.42

Coordinate occupancy of AP-1 sites in the vitamin D-responsive and CCAAT box elements by Fos-Jun in the osteocalcin gene: model for phenotype suppression of transcription. Proc Natl Acad Sci U S A (1990) 1.41

X-ray crystallographic analysis of the hydration of A- and B-form DNA at atomic resolution. Biopolymers (1998) 1.40

Mutant huntingtin expression in clonal striatal cells: dissociation of inclusion formation and neuronal survival by caspase inhibition. J Neurosci (1999) 1.40

Outer membrane of Escherichia coli K-12: differentiation of proteins 3A and 3B on acrylamide gels and further characterization of con (tolG) mutants. J Bacteriol (1976) 1.38

Pharmacokinetic properties of several novel oligonucleotide analogs in mice. J Pharmacol Exp Ther (1996) 1.38

Characterization of lethal zygosis associated with conjugation in Escherichia coli K-12. J Bacteriol (1973) 1.36

Molecular cloning of the tolC locus of Escherichia coli K-12 with the use of transposon Tn10. Mol Gen Genet (1981) 1.29

Cross-resistance between bacteriophages and colicins in Escherichia coli K-12. J Bacteriol (1976) 1.28

In vivo fate of phosphorothioate antisense oligodeoxynucleotides: predominant uptake by scavenger receptors on endothelial liver cells. Nucleic Acids Res (1997) 1.28

Immunohistochemical localization of the D1 dopamine receptor in rat brain reveals its axonal transport, pre- and postsynaptic localization, and prevalence in the basal ganglia, limbic system, and thalamic reticular nucleus. Proc Natl Acad Sci U S A (1992) 1.27

Limitations of biopsy Gleason grade: implications for counseling patients with biopsy Gleason score 6 prostate cancer. J Urol (2004) 1.27

Mode of action of colicins of types E1, E2, E3, and K. J Bacteriol (1968) 1.26

Therapeutic efficiency and safety of a second-generation replication-conditional HSV1 vector for brain tumor gene therapy. Hum Gene Ther (1997) 1.26

Gene expression. RNA binding: beta s and basics. Nature (1990) 1.23

Phosphorothioate oligodeoxyribonucleotides dissociate from cationic lipids before entering the nucleus. Nucleic Acids Res (1998) 1.22

Murine pharmacokinetics and antitumor efficacy of the photodynamic sensitizer 2-[1-hexyloxyethyl]-2-devinyl pyropheophorbide-a. J Photochem Photobiol B (1993) 1.21

Expression of normal and mutant huntingtin in the developing brain. J Neurosci (1996) 1.21

Operative management of acetabular fractures in Oxford. Injury (2001) 1.21

Lymphocyte chromosome analysis of agricultural workers during extensive occupational exposure to pesticides. Mutat Res (1973) 1.21

Fast transport and retrograde movement of huntingtin and HAP 1 in axons. Neuroreport (1997) 1.20

Synthesis, photophysical properties, tumor uptake, and preliminary in vivo photosensitizing efficacy of a homologous series of 3-(1'-alkyloxy)ethyl-3-devinylpurpurin-18-N-alkylimides with variable lipophilicity. J Med Chem (2001) 1.17

RNA annealing activity is intrinsically associated with U2AF. J Biol Chem (1993) 1.17

Are neuronal intranuclear inclusions the common neuropathology of triplet-repeat disorders with polyglutamine-repeat expansions? Lancet (1998) 1.17