| review article | Q7318358 |
| scholarly article | Q13442814 |
| P356 | DOI | 10.1093/NAR/GKU385 |
| P8608 | Fatcat ID | release_3lg5etwo3fhhhlu6cyki6cywii |
| P932 | PMC publication ID | 4066792 |
| P698 | PubMed publication ID | 24848018 |
| P5875 | ResearchGate publication ID | 262532312 |
| P50 | author | Włodzimierz Krzyżosiak | Q9382702 |
| Agnieszka Fiszer | Q59817047 | ||
| P2860 | cites work | Huntingtin interacting proteins are genetic modifiers of neurodegeneration | Q21145230 |
| Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy | Q24290177 | ||
| Nuclear localization or inclusion body formation of ataxin-2 are not necessary for SCA2 pathogenesis in mouse or human | Q24290184 | ||
| Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7 | Q24295641 | ||
| TRBP recruits the Dicer complex to Ago2 for microRNA processing and gene silencing | Q24306121 | ||
| Cloning of the SCA7 gene reveals a highly unstable CAG repeat expansion | Q24310393 | ||
| C-termini of P/Q-type Ca2+ channel alpha1A subunits translocate to nuclei and promote polyglutamine-mediated toxicity | Q24314234 | ||
| RORalpha-mediated Purkinje cell development determines disease severity in adult SCA1 mice | Q24316105 | ||
| Cloning of the gene for spinocerebellar ataxia 2 reveals a locus with high sensitivity to expanded CAG/glutamine repeats | Q24317096 | ||
| MicroRNAs: target recognition and regulatory functions | Q24609584 | ||
| Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study | Q24616493 | ||
| A natural antisense transcript at the Huntington's disease repeat locus regulates HTT expression | Q24620388 | ||
| Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease | Q24626259 | ||
| Towards a transgenic model of Huntington's disease in a non-human primate | Q24644013 | ||
| CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup | Q24644358 | ||
| Most mammalian mRNAs are conserved targets of microRNAs | Q24655061 | ||
| A neurological disease caused by an expanded CAG trinucleotide repeat in the TATA-binding protein gene: a new polyglutamine disease? | Q55670781 | ||
| Homozygous inheritance of the Machado-Joseph disease gene | Q57419142 | ||
| Analysis of the role of heat shock protein (Hsp) molecular chaperones in polyglutamine disease | Q73212216 | ||
| CUG repeats present in myotonin kinase RNA form metastable "slippery" hairpins | Q73919886 | ||
| Ribonuclease dicer cleaves triplet repeat hairpins into shorter repeats that silence specific targets | Q79818012 | ||
| A toxic monomeric conformer of the polyglutamine protein | Q79976767 | ||
| Imperfect CAG repeats form diverse structures in SCA1 transcripts | Q80413634 | ||
| Inclusion formation by ataxins -1, -2, -3, and -7 | Q80677124 | ||
| CAG repeats containing CAA interruptions form branched hairpin structures in spinocerebellar ataxia type 2 transcripts | Q80980223 | ||
| Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA | Q81547115 | ||
| Widespread suppression of huntingtin with convection-enhanced delivery of siRNA | Q82690762 | ||
| Reversibility of symptoms in a conditional mouse model of spinocerebellar ataxia type 3 | Q84377636 | ||
| Single-stranded siRNAs activate RNAi in animals | Q84913472 | ||
| Polyalanine and polyserine frameshift products in Huntington's disease | Q24656119 | ||
| Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells | Q24658337 | ||
| A systematic analysis of the silencing effects of an active siRNA at all single-nucleotide mismatched target sites | Q24804835 | ||
| Glutamine-expanded ataxin-7 alters TFTC/STAGA recruitment and chromatin structure leading to photoreceptor dysfunction. | Q25256616 | ||
| Mouse models of polyglutamine diseases: review and data table. Part I | Q26853025 | ||
| The panorama of miRNA-mediated mechanisms in mammalian cells | Q27001268 | ||
| Modeling Huntington's disease with induced pluripotent stem cells | Q27004209 | ||
| Mouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II. | Q27005950 | ||
| Polyglutamine neurodegeneration: expanded glutamines enhance native functions | Q27026773 | ||
| Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice | Q27324749 | ||
| Inhibition of alphavirus infection in cell culture and in mice with antisense morpholino oligomers | Q27486413 | ||
| Structural insights into CUG repeats containing the 'stretched U-U wobble': implications for myotonic dystrophy | Q27655444 | ||
| Atomic resolution structure of CAG RNA repeats: structural insights and implications for the trinucleotide repeat expansion diseases | Q27664034 | ||
| Myotonic Dystrophy Type 1 RNA Crystal Structures Reveal Heterogeneous 1 × 1 Nucleotide UU Internal Loop Conformations | Q27674953 | ||
| A dynamic structural model of expanded RNA CAG repeats: a refined X-ray structure and computational investigations using molecular dynamics and umbrella sampling simulations | Q27676633 | ||
| Protein-protein interactions as a strategy towards protein-specific drug design: the example of ataxin-1 | Q27680453 | ||
| Mammalian microRNAs predominantly act to decrease target mRNA levels | Q27860535 | ||
| RNA interference is mediated by 21- and 22-nucleotide RNAs | Q27860764 | ||
| Role for a bidentate ribonuclease in the initiation step of RNA interference | Q27860832 | ||
| A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. | Q27860836 | ||
| Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells | Q27860926 | ||
| RNAi factors are present and active in human cell nuclei | Q28111715 | ||
| Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells | Q28215061 | ||
| CAG expansions in a novel gene for Machado-Joseph disease at chromosome 14q32.1 | Q28235526 | ||
| In vivo genome editing restores haemostasis in a mouse model of haemophilia | Q28241725 | ||
| Highly efficient endogenous human gene correction using designed zinc-finger nucleases | Q28243157 | ||
| Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation | Q28246858 | ||
| Dentatorubral and pallidoluysian atrophy expansion of an unstable CAG trinucleotide on chromosome 12p | Q28250966 | ||
| Unstable expansion of CAG repeat in hereditary dentatorubral-pallidoluysian atrophy (DRPLA) | Q28250990 | ||
| Argonaute-1 directs siRNA-mediated transcriptional gene silencing in human cells | Q28260424 | ||
| Involvement of AGO1 and AGO2 in mammalian transcriptional silencing | Q28260436 | ||
| Caspase cleavage of gene products associated with triplet expansion disorders generates truncated fragments containing the polyglutamine tract | Q28267110 | ||
| RNA toxicity is a component of ataxin-3 degeneration in Drosophila | Q28278448 | ||
| RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform | Q34020786 | ||
| Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy | Q34023072 | ||
| SCA17, a novel autosomal dominant cerebellar ataxia caused by an expanded polyglutamine in TATA-binding protein | Q34083719 | ||
| The discovery of zinc fingers and their applications in gene regulation and genome manipulation. | Q34101354 | ||
| Chemical correction of pre-mRNA splicing defects associated with sequestration of muscleblind-like 1 protein by expanded r(CAG)-containing transcripts | Q34129996 | ||
| Small interfering RNA-induced transcriptional gene silencing in human cells | Q34161327 | ||
| Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs | Q34163091 | ||
| Homozygosity for CAG mutation in Huntington disease is associated with a more severe clinical course | Q34181024 | ||
| A pathogenic mechanism in Huntington's disease involves small CAG-repeated RNAs with neurotoxic activity. | Q34181368 | ||
| An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases | Q34187525 | ||
| Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin | Q34221351 | ||
| RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1 | Q34235172 | ||
| RNA therapeutics: beyond RNA interference and antisense oligonucleotides | Q34248493 | ||
| Human RNase H1 Uses One Tryptophan and Two Lysines to Position the Enzyme at the 3′-DNA/5′-RNA Terminus of the Heteroduplex Substrate | Q34264646 | ||
| Huntingtin protein interactions altered by polyglutamine expansion as determined by quantitative proteomic analysis. | Q34274478 | ||
| Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. | Q34283652 | ||
| Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs | Q34298362 | ||
| Advances in therapeutic RNA-targeting | Q34325519 | ||
| Structural requirements at the catalytic site of the heteroduplex substrate for human RNase H1 catalysis | Q34327729 | ||
| Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat | Q34348297 | ||
| Expansion of an unstable trinucleotide CAG repeat in spinocerebellar ataxia type 1. | Q34353488 | ||
| Gene silencing using micro-RNA designed hairpins | Q34364532 | ||
| Moderate expansion of a normally biallelic trinucleotide repeat in spinocerebellar ataxia type 2. | Q34405223 | ||
| Identification of the spinocerebellar ataxia type 2 gene using a direct identification of repeat expansion and cloning technique, DIRECT. | Q34405231 | ||
| Therapeutic approaches to preventing cell death in Huntington disease | Q34411338 | ||
| Tailor-made RNAi knockdown against triplet repeat disease-causing alleles | Q34411459 | ||
| Full motor recovery despite striatal neuron loss and formation of irreversible amyloid-like inclusions in a conditional mouse model of Huntington's disease. | Q34461236 | ||
| Non-ATG-initiated translation directed by microsatellite expansions | Q34471662 | ||
| Mutant huntingtin: nuclear translocation and cytotoxicity mediated by GAPDH | Q34480379 | ||
| Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism | Q34492631 | ||
| Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference | Q34520619 | ||
| Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways | Q34530552 | ||
| RNA structure of trinucleotide repeats associated with human neurological diseases | Q34542108 | ||
| The pathogenic mechanisms of polyglutamine diseases and current therapeutic strategies | Q37570639 | ||
| Novel RNA-based strategies for therapeutic gene silencing | Q37678180 | ||
| Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice. | Q37696030 | ||
| HTT-lowering reverses Huntington's disease immune dysfunction caused by NFκB pathway dysregulation | Q37697482 | ||
| Kaleidoscopic protein-protein interactions in the life and death of ataxin-1: new strategies against protein aggregation | Q37706493 | ||
| Polyglutamine toxicity in non-neuronal cells | Q37709833 | ||
| Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease | Q37776087 | ||
| Huntington's disease: can mice lead the way to treatment? | Q37840353 | ||
| Cellular toxicity of expanded RNA repeats: focus on RNA foci | Q37897768 | ||
| Recent advances in understanding of the immunological off-target effects of siRNA. | Q37948943 | ||
| SCA1-phosphorylation, a regulator of Ataxin-1 function and pathogenesis | Q38005302 | ||
| Targeting mutant huntingtin for the development of disease-modifying therapy | Q38024634 | ||
| Advances in targeted genome editing | Q38028180 | ||
| Gene silencing by chemically modified siRNAs | Q38028308 | ||
| Exosomes for targeted siRNA delivery across biological barriers. | Q38037911 | ||
| Potential therapeutic applications of differentiated induced pluripotent stem cells (iPSCs) in the treatment of neurodegenerative diseases | Q38052340 | ||
| Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides. | Q38085679 | ||
| Silencing human genetic diseases with oligonucleotide-based therapies. | Q38089634 | ||
| Pharmacological protein targets in polyglutamine diseases: mutant polypeptides and their interactors | Q38107677 | ||
| Choosing an animal model for the study of Huntington's disease | Q38139868 | ||
| Pharmacokinetics of phosphorothioate antisense oligodeoxynucleotides | Q38296665 | ||
| Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon | Q38315639 | ||
| Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA. | Q38352202 | ||
| Neuronal properties, in vivo effects, and pathology of a Huntington's disease patient-derived induced pluripotent stem cells | Q38462208 | ||
| Trinucleotide repeats in human genome and exome. | Q38507135 | ||
| CAG repeats mimic CUG repeats in the misregulation of alternative splicing | Q38855978 | ||
| Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification | Q39038134 | ||
| Multifunctional receptor-targeted nanocomplexes for the delivery of therapeutic nucleic acids to the brain | Q39110042 | ||
| Identification of NUB1 as a suppressor of mutant Huntington toxicity via enhanced protein clearance | Q39176432 | ||
| Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells | Q39303125 | ||
| Quantitative proteomic analysis of induced pluripotent stem cells derived from a human Huntington's disease patient | Q39331956 | ||
| The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity | Q39382468 | ||
| Spt4 is selectively required for transcription of extended trinucleotide repeats | Q39395626 | ||
| Perturbation of U2AF65/NXF1-mediated RNA nuclear export enhances RNA toxicity in polyQ diseases | Q39513867 | ||
| Mutant CAG repeats of Huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference | Q39605747 | ||
| Human RISC couples microRNA biogenesis and posttranscriptional gene silencing | Q28280424 | ||
| Genome editing with engineered zinc finger nucleases | Q28290795 | ||
| Autosomal dominant cerebellar ataxia (SCA6) associated with small polyglutamine expansions in the alpha 1A-voltage-dependent calcium channel | Q28300848 | ||
| Phosphorylation of S776 and 14-3-3 binding modulate ataxin-1 interaction with splicing factors | Q28472246 | ||
| Characterization of Human Huntington's Disease Cell Model from Induced Pluripotent Stem Cells | Q28475963 | ||
| Neurons lacking huntingtin differentially colonize brain and survive in chimeric mice | Q28511460 | ||
| Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease | Q28588314 | ||
| Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice | Q28594828 | ||
| Specificity of microRNA target selection in translational repression | Q28776123 | ||
| Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing | Q28830393 | ||
| Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs | Q28914781 | ||
| Trinucleotide Repeat Disorders | Q29038716 | ||
| Regulation of mRNA translation and stability by microRNAs | Q29547304 | ||
| Rational siRNA design for RNA interference | Q29614450 | ||
| Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases | Q29615069 | ||
| Expression profiling reveals off-target gene regulation by RNAi | Q29615965 | ||
| Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes | Q29617554 | ||
| Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain | Q29617982 | ||
| New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats | Q30427374 | ||
| A fully humanized transgenic mouse model of Huntington disease. | Q30537951 | ||
| Biologically active molecules that reduce polyglutamine aggregation and toxicity | Q33244412 | ||
| Designing siRNA that distinguish between genes that differ by a single nucleotide | Q33257088 | ||
| Interaction of Akt-phosphorylated ataxin-1 with 14-3-3 mediates neurodegeneration in spinocerebellar ataxia type 1. | Q33292483 | ||
| Polyglutamine neurodegenerative diseases and regulation of transcription: assembling the puzzle | Q33343058 | ||
| Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease | Q33374523 | ||
| Rational and modular design of potent ligands targeting the RNA that causes myotonic dystrophy 2. | Q33427148 | ||
| Small molecule drug discovery for Huntington's Disease | Q33442685 | ||
| Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3. | Q33474085 | ||
| Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype. | Q33507519 | ||
| Sequence-non-specific effects of RNA interference triggers and microRNA regulators | Q33566425 | ||
| Controlling the specificity of modularly assembled small molecules for RNA via ligand module spacing: targeting the RNAs that cause myotonic muscular dystrophy | Q33569231 | ||
| Asymmetric shorter-duplex siRNA structures trigger efficient gene silencing with reduced nonspecific effects | Q33713446 | ||
| Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. | Q33713689 | ||
| The role of flexibility in the rational design of modularly assembled ligands targeting the RNAs that cause the myotonic dystrophies | Q33763698 | ||
| Huntington's disease: from molecular pathogenesis to clinical treatment | Q33773061 | ||
| Long tract of untranslated CAG repeats is deleterious in transgenic mice | Q33808904 | ||
| An ovine transgenic Huntington's disease model | Q33815561 | ||
| Bidirectional expression of CUG and CAG expansion transcripts and intranuclear polyglutamine inclusions in spinocerebellar ataxia type 8. | Q34542422 | ||
| Ribosomal frameshifting on MJD-1 transcripts with long CAG tracts | Q34559976 | ||
| Drug properties of second-generation antisense oligonucleotides: how do they measure up to their predecessors? | Q34560139 | ||
| Structural MRI in Huntington's disease and recommendations for its potential use in clinical trials | Q34572794 | ||
| Small molecules enhance autophagy and reduce toxicity in Huntington's disease models. | Q34626231 | ||
| Designing highly active siRNAs for therapeutic applications. | Q34644554 | ||
| CAG repeat disorder models and human neuropathology: similarities and differences | Q34677190 | ||
| Off-target effects by siRNA can induce toxic phenotype | Q34703017 | ||
| Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi | Q34768688 | ||
| Ribonuclease H: the enzymes in eukaryotes | Q34948246 | ||
| Promoter targeted small RNAs induce long-term transcriptional gene silencing in human cells | Q34966942 | ||
| Antisense and antigene inhibition of gene expression by cell-permeable oligonucleotide-oligospermine conjugates. | Q35019418 | ||
| Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting | Q35030912 | ||
| Allele-specific silencing of dominant disease genes | Q35163317 | ||
| Ubiquitin-mediated sequestration of normal cellular proteins into polyglutamine aggregates | Q35171105 | ||
| Allele-specific silencing of mutant Huntington's disease gene | Q35194425 | ||
| The activity of siRNA in mammalian cells is related to structural target accessibility: a comparison with antisense oligonucleotides | Q35222789 | ||
| Genes and pathways affected by CAG-repeat RNA-based toxicity in Drosophila | Q35565702 | ||
| Stabilization of expanded (CTG)•(CAG) repeats by antisense oligonucleotides | Q35623474 | ||
| Triplet repeat RNA structure and its role as pathogenic agent and therapeutic target | Q35633648 | ||
| Expanding the action of duplex RNAs into the nucleus: redirecting alternative splicing | Q35740521 | ||
| Comparison of approaches for rational siRNA design leading to a new efficient and transparent method | Q35829289 | ||
| Argonaute and the nuclear RNAs: new pathways for RNA-mediated control of gene expression | Q35867143 | ||
| Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum | Q35888618 | ||
| Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates | Q35892068 | ||
| miRNA goes nuclear | Q36062602 | ||
| Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits | Q36089160 | ||
| CAG expansion induces nucleolar stress in polyglutamine diseases | Q36170710 | ||
| Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. | Q36239309 | ||
| Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice | Q36389503 | ||
| ROCK-phosphorylated vimentin modifies mutant huntingtin aggregation via sequestration of IRBIT. | Q36411728 | ||
| Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway | Q36477988 | ||
| Reduction of mutant ataxin-7 expression restores motor function and prevents cerebellar synaptic reorganization in a conditional mouse model of SCA7 | Q36580728 | ||
| Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease | Q36598263 | ||
| Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3? | Q39724719 | ||
| Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease | Q39795092 | ||
| Sustained effects of nonallele-specific Huntingtin silencing | Q39866098 | ||
| Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. | Q39913243 | ||
| Proteolytic cleavage of ataxin-7 by caspase-7 modulates cellular toxicity and transcriptional dysregulation | Q40103515 | ||
| Structural diversity of triplet repeat RNAs | Q40103612 | ||
| A systematic analysis of the effect of target RNA structure on RNA interference | Q40118209 | ||
| Design of siRNAs producing unstructured guide-RNAs results in improved RNA interference efficiency. | Q40356535 | ||
| New RNAi strategy for selective suppression of a mutant allele in polyglutamine disease | Q40359941 | ||
| Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. | Q40397746 | ||
| A structure-based analysis of huntingtin mutant polyglutamine aggregation and toxicity: evidence for a compact beta-sheet structure. | Q40462323 | ||
| Coupling of RNAi-mediated target downregulation with gene replacement | Q40638025 | ||
| Centrosome disorganization in fibroblast cultures derived from R6/2 Huntington's disease (HD) transgenic mice and HD patients | Q40771044 | ||
| Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer | Q41069577 | ||
| Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing | Q41627339 | ||
| Dynamic combinatorial selection of molecules capable of inhibiting the (CUG) repeat RNA-MBNL1 interaction in vitro: discovery of lead compounds targeting myotonic dystrophy (DM1). | Q41837351 | ||
| Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression | Q41898404 | ||
| Essential role of coiled coils for aggregation and activity of Q/N-rich prions and PolyQ proteins | Q41993338 | ||
| Effect of base modifications on structure, thermodynamic stability, and gene silencing activity of short interfering RNA. | Q42046598 | ||
| "Huntingtin holiday": progress toward an antisense therapy for Huntington's disease | Q42048629 | ||
| Aggregation of polyQ-extended proteins is promoted by interaction with their natural coiled-coil partners | Q42142509 | ||
| RNA interference-mediated suppression and replacement of human rhodopsin in vivo | Q42413344 | ||
| Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients | Q42559350 | ||
| Inhibiting gene expression at transcription start sites in chromosomal DNA with antigene RNAs | Q42677869 | ||
| Widespread expression of Huntington's disease gene (IT15) protein product | Q42684112 | ||
| Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes | Q42714345 | ||
| Induction of angiogenesis in a mouse model using engineered transcription factors | Q42808653 | ||
| Excitation-induced ataxin-3 aggregation in neurons from patients with Machado-Joseph disease | Q42818202 | ||
| Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats | Q42836546 | ||
| Whole gene expression profile in blood reveals multiple pathways deregulation in R6/2 mouse model | Q42918972 | ||
| Improved nucleic acid descriptors for siRNA efficacy prediction | Q43124137 | ||
| RNAimmuno: a database of the nonspecific immunological effects of RNA interference and microRNA reagents | Q43149714 | ||
| Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum | Q43781780 | ||
| The IGF-1/Akt pathway is neuroprotective in Huntington's disease and involves Huntingtin phosphorylation by Akt. | Q44025697 | ||
| High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo | Q44162261 | ||
| The impact of target site accessibility on the design of effective siRNAs | Q44175967 | ||
| Loss of wild-type huntingtin influences motor dysfunction and survival in the YAC128 mouse model of Huntington disease | Q44282485 | ||
| RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. | Q44964949 | ||
| Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. | Q45289792 | ||
| Blocking acid-sensing ion channel 1 alleviates Huntington's disease pathology via an ubiquitin-proteasome system-dependent mechanism | Q45290276 | ||
| A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference. | Q45293098 | ||
| Molecular architecture of CAG repeats in human disease related transcripts | Q45293998 | ||
| Patterns of CAG repeat interruptions in SCA1 and SCA2 genes in relation to repeat instability | Q45294379 | ||
| Huntingtin-encoded polyglutamine expansions form amyloid-like protein aggregates in vitro and in vivo | Q45294913 | ||
| Gene expression profile in fibroblasts of Huntington's disease patients and controls. | Q45296189 | ||
| Gene expression in Huntington's disease skeletal muscle: a potential biomarker | Q45296851 | ||
| Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein | Q45296862 | ||
| Cleavage at the caspase-6 site is required for neuronal dysfunction and degeneration due to mutant huntingtin | Q45301569 | ||
| Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice | Q45306356 | ||
| Chromosomal profiles of gene expression in Huntington's disease | Q45307320 | ||
| Specific and potent RNAi in the nucleus of human cells | Q45345569 | ||
| 2'-O-(2-Methoxy)ethyl-modified anti-intercellular adhesion molecule 1 (ICAM-1) oligonucleotides selectively increase the ICAM-1 mRNA level and inhibit formation of the ICAM-1 translation initiation complex in human umbilical vein endothelial cells. | Q46002868 | ||
| Inhibiting transcription of chromosomal DNA with antigene peptide nucleic acids. | Q46891008 | ||
| ATAXIN-1 interacts with the repressor Capicua in its native complex to cause SCA1 neuropathology | Q47072817 | ||
| Polyglutamine-expanded ataxin-7 inhibits STAGA histone acetyltransferase activity to produce retinal degeneration | Q33854083 | ||
| Development of biomarkers for Huntington's disease | Q33907629 | ||
| Ancestral origins of the Machado-Joseph disease mutation: a worldwide haplotype study | Q33929425 | ||
| RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model | Q33936969 | ||
| In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models | Q33951409 | ||
| Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3' end of a transcript encoding a protein kinase family member. | Q33968674 | ||
| Sequence-dependent and independent inhibition specific for mutant ataxin-3 by small interfering RNA. | Q47242742 | ||
| Duplication of Atxn1l suppresses SCA1 neuropathology by decreasing incorporation of polyglutamine-expanded ataxin-1 into native complexes | Q48081281 | ||
| Huntingtin acts in the nucleus to induce apoptosis but death does not correlate with the formation of intranuclear inclusions. | Q48373564 | ||
| The pathogenic agent in Drosophila models of 'polyglutamine' diseases | Q50280437 | ||
| Recovery from polyglutamine-induced neurodegeneration in conditional SCA1 transgenic mice. | Q52560509 | ||
| A Rapid Cellular FRET Assay of Polyglutamine Aggregation Identifies a Novel Inhibitor | Q52641586 | ||
| Ataxin-3 suppresses polyglutamine neurodegeneration in Drosophila by a ubiquitin-associated mechanism. | Q52656408 | ||
| Expanded ATXN3 frameshifting events are toxic in Drosophila and mammalian neuron models. | Q52734262 | ||
| Asymmetric RNA duplexes mediate RNA interference in mammalian cells. | Q53436494 | ||
| Translation of HTT mRNA with expanded CAG repeats is regulated by the MID1-PP2A protein complex. | Q54447316 | ||
| RNA learns from antisense | Q36685858 | ||
| Enhanced aggregation of androgen receptor in induced pluripotent stem cell-derived neurons from spinal and bulbar muscular atrophy | Q36708477 | ||
| Generation of human-induced pluripotent stem cells to model spinocerebellar ataxia type 2 in vitro | Q36717931 | ||
| RNA interference and innate immunity | Q36801842 | ||
| Huntington's disease protein contributes to RNA-mediated gene silencing through association with Argonaute and P bodies. | Q36825291 | ||
| Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs | Q36852787 | ||
| RNA toxicity in polyglutamine disorders: concepts, models, and progress of research | Q36859984 | ||
| Polyglutamine neurodegeneration: protein misfolding revisited. | Q36967107 | ||
| Gene therapy for misfolding protein diseases of the central nervous system | Q36983203 | ||
| Overcoming the innate immune response to small interfering RNA. | Q37071130 | ||
| Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice | Q37131550 | ||
| Impact of target mRNA structure on siRNA silencing efficiency: A large-scale study | Q37135903 | ||
| Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides | Q37193000 | ||
| Repeat-associated non-ATG (RAN) translation in neurological disease | Q37195032 | ||
| A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity | Q37199509 | ||
| RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression | Q37224701 | ||
| Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease | Q37254846 | ||
| Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice | Q37254850 | ||
| ss-siRNAs allele selectively inhibit ataxin-3 expression: multiple mechanisms for an alternative gene silencing strategy | Q37271206 | ||
| Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy. | Q37310657 | ||
| Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. | Q37327574 | ||
| Rational design of chemical genetic probes of RNA function and lead therapeutics targeting repeating transcripts | Q37360833 | ||
| A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding. | Q37364023 | ||
| Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs | Q37395054 | ||
| Pentamidine reverses the splicing defects associated with myotonic dystrophy. | Q37413602 | ||
| Post-translational modifications of expanded polyglutamine proteins: impact on neurotoxicity. | Q37417909 | ||
| Deciphering the code of innate immunity recognition of siRNAs | Q37419294 | ||
| Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides. | Q37419563 | ||
| Profiling of mismatch discrimination in RNAi enabled rational design of allele-specific siRNAs. | Q37477571 | ||
| Allele-selective inhibition of expression of huntingtin and ataxin-3 by RNA duplexes containing unlocked nucleic acid substitutions | Q37483574 | ||
| Huntington's disease: the current state of research with peripheral tissues. | Q37491501 | ||
| Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA. | Q37536671 | ||
| Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs | Q37536687 | ||
| P275 | copyright license | Creative Commons Attribution-NonCommercial 3.0 Unported | Q18810331 |
| P433 | issue | 11 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | genetic disease | Q200779 |
| genetic variation | Q349856 | ||
| regulation of gene expression | Q411391 | ||
| nucleotide | Q28745 | ||
| P5008 | on focus list of Wikimedia project | ScienceSource | Q55439927 |
| P304 | page(s) | 6787-6810 | |
| P577 | publication date | 2014-05-21 | |
| 2014-06-01 | |||
| P1433 | published in | Nucleic Acids Research | Q135122 |
| P1476 | title | Oligonucleotide-based strategies to combat polyglutamine diseases | |
| P478 | volume | 42 |
| Q42121340 | A brain-targeting lipidated peptide for neutralizing RNA-mediated toxicity in Polyglutamine Diseases. |
| Q47236927 | A peptidylic inhibitor for neutralizing expanded CAG RNA-induced nucleolar stress in polyglutamine diseases |
| Q28272901 | Activating frataxin expression by repeat-targeted nucleic acids |
| Q38547027 | Allele-selective suppression of mutant genes in polyglutamine diseases |
| Q54119194 | Association analysis of SNPs present in plasma with adverse events and population pharmacokinetics in Chinese sunitinib treated patients with renal cell carcinoma. |
| Q27028008 | Current understanding of the role of microRNAs in spinocerebellar ataxias |
| Q41490579 | Magnetic Nanoparticle Assisted Self-assembly of Cell Penetrating Peptides-Oligonucleotides Complexes for Gene Delivery. |
| Q39061798 | Molecular mechanisms underlying Spinocerebellar Ataxia 17 (SCA17) pathogenesis |
| Q37539531 | Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells |
| Q39112084 | Nanoparticulate strategies for the treatment of polyglutamine diseases by halting the protein aggregation process |
| Q38904908 | Non-coding RNAs as drug targets |
| Q26764958 | Precision medicine in spinocerebellar ataxias: treatment based on common mechanisms of disease |
| Q45735441 | Protein Misfolding and Aggregation as a Therapeutic Target for Polyglutamine Diseases |
| Q49679343 | RNA in the spotlight: the dawn of RNA therapeutics in the treatment of human disease |
| Q28082520 | RNA-mediated pathogenic mechanisms in polyglutamine diseases and amyotrophic lateral sclerosis |
| Q37723428 | Reduction of Huntington's Disease RNA Foci by CAG Repeat-Targeting Reagents |
| Q39256709 | Regulation of mRNA Translation by MID1: A Common Mechanism of Expanded CAG Repeat RNAs |
| Q91069266 | Spinocerebellar ataxias: prospects and challenges for therapy development |
| Q38994216 | Structures of RNA repeats associated with neurological diseases |
| Q38290349 | Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels. |
| Q36627956 | The first crystal structures of RNA-PNA duplexes and a PNA-PNA duplex containing mismatches--toward anti-sense therapy against TREDs |
| Q35271805 | The role of the immune system in triplet repeat expansion diseases |
| Q54262475 | Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic. |
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