Published in Neuron on May 12, 2011
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
CGG repeat-associated translation mediates neurodegeneration in fragile X tremor ataxia syndrome. Neuron (2013) 2.94
Human embryonic stem cell-derived GABA neurons correct locomotion deficits in quinolinic acid-lesioned mice. Cell Stem Cell (2012) 2.62
RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia. Proc Natl Acad Sci U S A (2013) 2.31
Cellular toxicity of expanded RNA repeats: focus on RNA foci. Hum Mol Genet (2011) 2.05
Epigenetic mechanisms in neurological disease. Nat Med (2012) 1.74
Neurodegeneration the RNA way. Prog Neurobiol (2011) 1.43
Physical chemistry of polyglutamine: intriguing tales of a monotonous sequence. J Mol Biol (2012) 1.37
Mechanisms of toxicity in C9FTLD/ALS. Acta Neuropathol (2014) 1.35
Triplet repeat RNA structure and its role as pathogenic agent and therapeutic target. Nucleic Acids Res (2011) 1.29
Slow amyloid nucleation via α-helix-rich oligomeric intermediates in short polyglutamine-containing huntingtin fragments. J Mol Biol (2011) 1.17
RNA-binding proteins in microsatellite expansion disorders: mediators of RNA toxicity. Brain Res (2012) 1.11
RNA-mediated toxicity in neurodegenerative disease. Mol Cell Neurosci (2012) 1.07
Inhibiting the nucleation of amyloid structure in a huntingtin fragment by targeting α-helix-rich oligomeric intermediates. J Mol Biol (2011) 1.03
Autophagy and polyglutamine diseases. Prog Neurobiol (2011) 1.02
Brain, blood, and iron: perspectives on the roles of erythrocytes and iron in neurodegeneration. Neurobiol Dis (2012) 0.99
Nucleotide excision repair, mismatch repair, and R-loops modulate convergent transcription-induced cell death and repeat instability. PLoS One (2012) 0.97
Accurate typing of short tandem repeats from genome-wide sequencing data and its applications. Genome Res (2015) 0.96
Serine phosphorylation suppresses huntingtin amyloid accumulation by altering protein aggregation properties. J Mol Biol (2012) 0.95
The DNAJB6 and DNAJB8 protein chaperones prevent intracellular aggregation of polyglutamine peptides. J Biol Chem (2013) 0.95
Kinetically competing huntingtin aggregation pathways control amyloid polymorphism and properties. Biochemistry (2012) 0.94
PolyQ disease: misfiring of a developmental cell death program? Trends Cell Biol (2012) 0.93
Mechanisms of RNA-induced toxicity in CAG repeat disorders. Cell Death Dis (2013) 0.91
Impaired pre-mRNA processing and altered architecture of 3' untranslated regions contribute to the development of human disorders. Int J Mol Sci (2013) 0.89
Neuroprotective effects of psychotropic drugs in Huntington's disease. Int J Mol Sci (2013) 0.88
Aggregation behavior of chemically synthesized, full-length huntingtin exon1. Biochemistry (2014) 0.87
Biophysical underpinnings of the repeat length dependence of polyglutamine amyloid formation. J Biol Chem (2014) 0.87
Brain pathology in myotonic dystrophy: when tauopathy meets spliceopathy and RNAopathy. Front Mol Neurosci (2014) 0.87
Expanded complexity of unstable repeat diseases. Biofactors (2012) 0.87
Hypertrophy in skeletal myotubes induced by junctophilin-2 mutant, Y141H, involves an increase in store-operated Ca2+ entry via Orai1. J Biol Chem (2012) 0.85
Acetyltransferases (HATs) as targets for neurological therapeutics. Neurotherapeutics (2013) 0.83
RNA imaging in living cells - methods and applications. RNA Biol (2014) 0.82
Bidirectional transcription of trinucleotide repeats: roles for excision repair. DNA Repair (Amst) (2013) 0.82
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
D-polyglutamine amyloid recruits L-polyglutamine monomers and kills cells. J Mol Biol (2013) 0.80
Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity. Hum Mol Genet (2014) 0.80
The junctophilin family of proteins: from bench to bedside. Trends Mol Med (2014) 0.80
Role of noncoding RNAs in trinucleotide repeat neurodegenerative disorders. Exp Neurol (2012) 0.79
Junctophilin 3 (JPH3) expansion mutations causing Huntington disease like 2 (HDL2) are common in South African patients with African ancestry and a Huntington disease phenotype. Am J Med Genet B Neuropsychiatr Genet (2015) 0.78
Ubiquitous expression of CUG or CAG trinucleotide repeat RNA causes common morphological defects in a Drosophila model of RNA-mediated pathology. PLoS One (2012) 0.78
Macrocerebellum, epilepsy, intellectual disability, and gut malrotation in a child with a 16q24.1-q24.2 contiguous gene deletion. Am J Med Genet A (2014) 0.78
Examination of Huntington's disease in a Chinese family. Neural Regen Res (2014) 0.77
RNA-binding protein misregulation in microsatellite expansion disorders. Adv Exp Med Biol (2014) 0.77
Update on the Non-Huntington's Disease Choreas with Comments on the Current Nomenclature. Tremor Other Hyperkinet Mov (N Y) (2012) 0.76
Mismatch repair enhances convergent transcription-induced cell death at trinucleotide repeats by activating ATR. DNA Repair (Amst) (2016) 0.76
Transcriptional maturation of the mouse auditory forebrain. BMC Genomics (2015) 0.76
Neuropathology and Cellular Pathogenesis of Spinocerebellar Ataxia Type 12. Mov Disord (2015) 0.75
Fanconi anemia pathway regulates convergent transcription-induced cell death at trinucleotide repeats in human cells. Postdoc J (2016) 0.75
Clinical and molecular research of neuroacanthocytosis. Neural Regen Res (2013) 0.75
RNA toxicity and foci formation in microsatellite expansion diseases. Curr Opin Genet Dev (2017) 0.75
Are polyglutamine diseases expanding? Neuron (2011) 0.75
A Drosophila model of Huntington disease-like 2 exhibits nuclear toxicity and distinct pathogenic mechanisms from Huntington disease. Hum Mol Genet (2016) 0.75
Backbone engineering within a latent β-hairpin structure to design inhibitors of polyglutamine amyloid formation. J Mol Biol (2016) 0.75
RNA-binding proteins in neurodegeneration: mechanisms in aggregate. Genes Dev (2017) 0.75
Generalized lacZ expression with the ROSA26 Cre reporter strain. Nat Genet (1999) 55.63
Cre reporter strains produced by targeted insertion of EYFP and ECFP into the ROSA26 locus. BMC Dev Biol (2001) 31.56
Antisense transcription in the mammalian transcriptome. Science (2005) 15.69
Trinucleotide repeat disorders. Annu Rev Neurosci (2007) 9.45
DNA methylation-related chromatin remodeling in activity-dependent BDNF gene regulation. Science (2003) 8.81
Histone deacetylase inhibitors arrest polyglutamine-dependent neurodegeneration in Drosophila. Nature (2001) 6.76
Homologous recombination based modification in Escherichia coli and germline transmission in transgenic mice of a bacterial artificial chromosome. Nat Biotechnol (1997) 6.29
A muscleblind knockout model for myotonic dystrophy. Science (2003) 5.93
p300/CBP proteins: HATs for transcriptional bridges and scaffolds. J Cell Sci (2001) 5.74
Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease. Science (2001) 5.28
RNA-mediated neuromuscular disorders. Annu Rev Neurosci (2006) 4.24
Interference by huntingtin and atrophin-1 with cbp-mediated transcription leading to cellular toxicity. Science (2001) 4.13
The Huntington's disease protein interacts with p53 and CREB-binding protein and represses transcription. Proc Natl Acad Sci U S A (2000) 4.01
Bidirectional expression of CUG and CAG expansion transcripts and intranuclear polyglutamine inclusions in spinocerebellar ataxia type 8. Nat Genet (2006) 3.87
Molecular mechanisms and potential therapeutical targets in Huntington's disease. Physiol Rev (2010) 3.69
Highly efficient modification of bacterial artificial chromosomes (BACs) using novel shuttle vectors containing the R6Kgamma origin of replication. Genome Res (2002) 3.61
Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci (2008) 3.54
A biological function for the neuronal activity-dependent component of Bdnf transcription in the development of cortical inhibition. Neuron (2008) 3.40
Insoluble detergent-resistant aggregates form between pathological and nonpathological lengths of polyglutamine in mammalian cells. Proc Natl Acad Sci U S A (1999) 3.33
Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2. Hum Mol Genet (2001) 3.27
Polyglutamine expansion as a pathological epitope in Huntington's disease and four dominant cerebellar ataxias. Nature (1995) 2.92
Serines 13 and 16 are critical determinants of full-length human mutant huntingtin induced disease pathogenesis in HD mice. Neuron (2009) 2.43
CREB-binding protein sequestration by expanded polyglutamine. Hum Mol Genet (2000) 2.18
Huntington's disease--like 2 is associated with CUG repeat-containing RNA foci. Ann Neurol (2007) 1.61
A repeat expansion in the gene encoding junctophilin-3 is associated with Huntington disease-like 2. Nat Genet (2001) 1.56
Aberrant histone acetylation, altered transcription, and retinal degeneration in a Drosophila model of polyglutamine disease are rescued by CREB-binding protein. Genes Dev (2003) 1.33
A comparison of huntington disease and huntington disease-like 2 neuropathology. J Neuropathol Exp Neurol (2008) 1.24
Huntingtin inclusions do not deplete polyglutamine-containing transcription factors in HD mice. Hum Mol Genet (2002) 1.14
Using antibodies to analyze polyglutamine stretches. Methods Mol Biol (2004) 1.14
Imaging polyglutamine deposits in brain tissue. Methods Enzymol (2006) 1.13
Mouse and fly models of neurodegeneration. Trends Genet (2002) 1.10
Huntington's Disease-like 2 (HDL2) in North America and Japan. Ann Neurol (2004) 1.01
A disorder similar to Huntington's disease is associated with a novel CAG repeat expansion. Ann Neurol (2001) 1.00
Huntington's disease like-2 neuropathology. Mov Disord (2007) 0.99
Huntington's disease like-2: review and update. Acta Neurol Taiwan (2005) 0.97
Motor discoordination in mutant mice lacking junctophilin type 3. Biochem Biophys Res Commun (2002) 0.97
Strong aggregation and increased toxicity of polyleucine over polyglutamine stretches in mammalian cells. Hum Mol Genet (2002) 0.97
The Huntington's disease-like syndromes: what to consider in patients with a negative Huntington's disease gene test. Nat Clin Pract Neurol (2007) 0.94
Diminished activity-dependent brain-derived neurotrophic factor expression underlies cortical neuron microcircuit hypoconnectivity resulting from exposure to mutant huntingtin fragments. J Pharmacol Exp Ther (2010) 0.91
The diploid genome sequence of an Asian individual. Nature (2008) 46.29
Guidelines for the use and interpretation of assays for monitoring autophagy. Autophagy (2012) 20.08
The sequence and de novo assembly of the giant panda genome. Nature (2009) 15.76
Modelling disease outbreaks in realistic urban social networks. Nature (2004) 14.54
Biosensing with plasmonic nanosensors. Nat Mater (2008) 12.10
DNA methylation-related chromatin remodeling in activity-dependent BDNF gene regulation. Science (2003) 8.81
The genome of the cucumber, Cucumis sativus L. Nat Genet (2009) 8.19
Ancient human genome sequence of an extinct Palaeo-Eskimo. Nature (2010) 7.51
Raltegravir with optimized background therapy for resistant HIV-1 infection. N Engl J Med (2008) 7.33
Subgroup and resistance analyses of raltegravir for resistant HIV-1 infection. N Engl J Med (2008) 5.58
Antiretroviral activity, pharmacokinetics, and tolerability of MK-0518, a novel inhibitor of HIV-1 integrase, dosed as monotherapy for 10 days in treatment-naive HIV-1-infected individuals. J Acquir Immune Defic Syndr (2006) 4.71
ATP-binding cassette transporters and HDL suppress hematopoietic stem cell proliferation. Science (2010) 4.67
HDL, ABC transporters, and cholesterol efflux: implications for the treatment of atherosclerosis. Cell Metab (2008) 4.34
Combined deficiency of ABCA1 and ABCG1 promotes foam cell accumulation and accelerates atherosclerosis in mice. J Clin Invest (2007) 3.89
FACS-array profiling of striatal projection neuron subtypes in juvenile and adult mouse brains. Nat Neurosci (2006) 3.81
Neurobiology of schizophrenia. Neuron (2006) 3.69
Probing the dynamics of O-GlcNAc glycosylation in the brain using quantitative proteomics. Nat Chem Biol (2007) 3.60
Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci (2008) 3.54
The cell and molecular basis of mechanical, cold, and inflammatory pain. Science (2008) 3.46
Thermoregulatory and metabolic defects in Huntington's disease transgenic mice implicate PGC-1alpha in Huntington's disease neurodegeneration. Cell Metab (2006) 3.38
Weight gain in the first week of life and overweight in adulthood: a cohort study of European American subjects fed infant formula. Circulation (2005) 3.28
A novel cyclosporine a drug-delivery system for prevention of human corneal rejection after high-risk keratoplasty: a clinical study. Ophthalmology (2012) 3.24
Activation of p53 by MEG3 non-coding RNA. J Biol Chem (2007) 3.07
Genetic programs in human and mouse early embryos revealed by single-cell RNA sequencing. Nature (2013) 3.06
Bergmann glia expression of polyglutamine-expanded ataxin-7 produces neurodegeneration by impairing glutamate transport. Nat Neurosci (2006) 3.05
Histone modifications around individual BDNF gene promoters in prefrontal cortex are associated with extinction of conditioned fear. Learn Mem (2007) 2.99
Increased inflammatory gene expression in ABC transporter-deficient macrophages: free cholesterol accumulation, increased signaling via toll-like receptors, and neutrophil infiltration of atherosclerotic lesions. Circulation (2008) 2.98
Control of PERK eIF2alpha kinase activity by the endoplasmic reticulum stress-induced molecular chaperone P58IPK. Proc Natl Acad Sci U S A (2002) 2.93
AgBase: a unified resource for functional analysis in agriculture. Nucleic Acids Res (2006) 2.92
Complete resequencing of 40 genomes reveals domestication events and genes in silkworm (Bombyx). Science (2009) 2.68
Raltegravir once daily or twice daily in previously untreated patients with HIV-1: a randomised, active-controlled, phase 3 non-inferiority trial. Lancet Infect Dis (2011) 2.66
Role of HDL, ABCA1, and ABCG1 transporters in cholesterol efflux and immune responses. Arterioscler Thromb Vasc Biol (2009) 2.64
Integrative genomic and functional analyses reveal neuronal subtype differentiation bias in human embryonic stem cell lines. Proc Natl Acad Sci U S A (2007) 2.60
A combinatorial F box protein directed pathway controls TRAF adaptor stability to regulate inflammation. Nat Immunol (2013) 2.60
A selective TrkB agonist with potent neurotrophic activities by 7,8-dihydroxyflavone. Proc Natl Acad Sci U S A (2010) 2.57
AgBase: a functional genomics resource for agriculture. BMC Genomics (2006) 2.53
Psychopathology in patients with degenerative cerebellar diseases: a comparison to Huntington's disease. Am J Psychiatry (2002) 2.52
Androgen receptor YAC transgenic mice recapitulate SBMA motor neuronopathy and implicate VEGF164 in the motor neuron degeneration. Neuron (2004) 2.52
ALS motor phenotype heterogeneity, focality, and spread: deconstructing motor neuron degeneration. Neurology (2009) 2.47
Pancreatic beta-cell failure and diabetes in mice with a deletion mutation of the endoplasmic reticulum molecular chaperone gene P58IPK. Diabetes (2005) 2.46
Serines 13 and 16 are critical determinants of full-length human mutant huntingtin induced disease pathogenesis in HD mice. Neuron (2009) 2.43
The roles of different pathways in the release of cholesterol from macrophages. J Lipid Res (2007) 2.38
Raltegravir versus Efavirenz regimens in treatment-naive HIV-1-infected patients: 96-week efficacy, durability, subgroup, safety, and metabolic analyses. J Acquir Immune Defic Syndr (2010) 2.37
CD133+ neural stem cells in the ependyma of mammalian postnatal forebrain. Proc Natl Acad Sci U S A (2008) 2.36
HDL from CETP-deficient subjects shows enhanced ability to promote cholesterol efflux from macrophages in an apoE- and ABCG1-dependent pathway. J Clin Invest (2006) 2.36
Genome-wide association identifies a susceptibility locus for coronary artery disease in the Chinese Han population. Nat Genet (2011) 2.32
Genetic variation in SCN10A influences cardiac conduction. Nat Genet (2010) 2.30
A positive autoregulatory loop of Jak-STAT signaling controls the onset of astrogliogenesis. Nat Neurosci (2005) 2.30
Systematic behavioral evaluation of Huntington's disease transgenic and knock-in mouse models. Neurobiol Dis (2009) 2.29
Decreased atherosclerosis in low-density lipoprotein receptor knockout mice transplanted with Abcg1-/- bone marrow. Arterioscler Thromb Vasc Biol (2006) 2.29
Associations between single-nucleotide polymorphisms in the PI3K-PTEN-AKT-mTOR pathway and increased risk of brain metastasis in patients with non-small cell lung cancer. Clin Cancer Res (2013) 2.28
Synthesis, molecular modeling, and biological evaluation of cinnamic acid metronidazole ester derivatives as novel anticancer agents. Bioorg Med Chem (2010) 2.25
Identifying polyglutamine protein species in situ that best predict neurodegeneration. Nat Chem Biol (2011) 2.24
National study of US emergency department visits for attempted suicide and self-inflicted injury, 1997-2001. Ann Emerg Med (2005) 2.20
Activation of the indole-3-acetic acid-amido synthetase GH3-8 suppresses expansin expression and promotes salicylate- and jasmonate-independent basal immunity in rice. Plant Cell (2008) 2.19
High-density lipoprotein protects macrophages from oxidized low-density lipoprotein-induced apoptosis by promoting efflux of 7-ketocholesterol via ABCG1. Proc Natl Acad Sci U S A (2007) 2.18
ABCG1 and HDL protect against endothelial dysfunction in mice fed a high-cholesterol diet. J Clin Invest (2008) 2.16
Long-term efficacy and safety of Raltegravir combined with optimized background therapy in treatment-experienced patients with drug-resistant HIV infection: week 96 results of the BENCHMRK 1 and 2 Phase III trials. Clin Infect Dis (2010) 2.14
Independent validation of candidate breast cancer serum biomarkers identified by mass spectrometry. Clin Chem (2005) 2.13
Pathological cell-cell interactions elicited by a neuropathogenic form of mutant Huntingtin contribute to cortical pathogenesis in HD mice. Neuron (2005) 2.11
H2S generated by heart in rat and its effects on cardiac function. Biochem Biophys Res Commun (2004) 2.11
Proteolysis of mutant huntingtin produces an exon 1 fragment that accumulates as an aggregated protein in neuronal nuclei in Huntington disease. J Biol Chem (2010) 2.10
Dispersive pad site burns with modern radiofrequency ablation equipment. Surg Laparosc Endosc Percutan Tech (2003) 2.09
DNA methylation controls the timing of astrogliogenesis through regulation of JAK-STAT signaling. Development (2005) 2.08
PGC-1α rescues Huntington's disease proteotoxicity by preventing oxidative stress and promoting TFEB function. Sci Transl Med (2012) 2.07
Regulation of IRF-3-dependent innate immunity by the papain-like protease domain of the severe acute respiratory syndrome coronavirus. J Biol Chem (2007) 2.07
c-Myc-mediated epigenetic silencing of MicroRNA-101 contributes to dysregulation of multiple pathways in hepatocellular carcinoma. Hepatology (2014) 2.04
Efficacy, safety, and immunogenicity of an enterovirus 71 vaccine in China. N Engl J Med (2014) 2.01
Improving penetration in tumors with nanoassemblies of phospholipids and doxorubicin. J Natl Cancer Inst (2007) 2.00
Early autophagic response in a novel knock-in model of Huntington disease. Hum Mol Genet (2010) 1.99
Activity-dependent synaptic Wnt release regulates hippocampal long term potentiation. J Biol Chem (2006) 1.98
Endogenous hydrogen sulfide regulation of myocardial injury induced by isoproterenol. Biochem Biophys Res Commun (2004) 1.98
Neuroprotective role of Sirt1 in mammalian models of Huntington's disease through activation of multiple Sirt1 targets. Nat Med (2011) 1.97
A model to predict antiviral treatment in HBeAg negative chronic hepatitis B with alanine aminotransferase≤2 upper limit of normal. Liver Int (2013) 1.97
Polyglutamine-expanded ataxin-7 inhibits STAGA histone acetyltransferase activity to produce retinal degeneration. Proc Natl Acad Sci U S A (2005) 1.97
A novel copper complex of salicylaldehyde pyrazole hydrazone induces apoptosis through up-regulating integrin beta4 in H322 lung carcinoma cells. Eur J Med Chem (2010) 1.94
Loss of endogenous androgen receptor protein accelerates motor neuron degeneration and accentuates androgen insensitivity in a mouse model of X-linked spinal and bulbar muscular atrophy. Hum Mol Genet (2006) 1.93
Purkinje cell degeneration (pcd) phenotypes caused by mutations in the axotomy-induced gene, Nna1. Science (2002) 1.90
The divergent DSL ligand Dll3 does not activate Notch signaling but cell autonomously attenuates signaling induced by other DSL ligands. J Cell Biol (2005) 1.88
Coupling of cell migration with neurogenesis by proneural bHLH factors. Proc Natl Acad Sci U S A (2006) 1.87
Effects of 1-H-indole-3-glyoxamide (A-002) on concentration of secretory phospholipase A2 (PLASMA study): a phase II double-blind, randomised, placebo-controlled trial. Lancet (2009) 1.87
A genome scan for modifiers of age at onset in Huntington disease: The HD MAPS study. Am J Hum Genet (2003) 1.85
MicroRNA-204/211 alters epithelial physiology. FASEB J (2010) 1.83
US emergency department visits for alcohol-related diseases and injuries between 1992 and 2000. Arch Intern Med (2004) 1.82
Quantification of O-glycosylation stoichiometry and dynamics using resolvable mass tags. Nat Chem Biol (2010) 1.79
Controlled plasmonic nanostructures for surface-enhanced spectroscopy and sensing. Acc Chem Res (2008) 1.79
A phenotypic small-molecule screen identifies an orphan ligand-receptor pair that regulates neural stem cell differentiation. Chem Biol (2007) 1.78
Roles of glutamate receptors and the mammalian target of rapamycin (mTOR) signaling pathway in activity-dependent dendritic protein synthesis in hippocampal neurons. J Biol Chem (2006) 1.77
Long-term treatment with raltegravir or efavirenz combined with tenofovir/emtricitabine for treatment-naive human immunodeficiency virus-1-infected patients: 156-week results from STARTMRK. Clin Infect Dis (2011) 1.77
Evaluation of refractive error measurements of the Wavescan Wavefront system and the Tracey Wavefront aberrometer. J Cataract Refract Surg (2003) 1.76
Regulation and mechanisms of macrophage cholesterol efflux. J Clin Invest (2002) 1.76