| scholarly article | Q13442814 |
| P50 | author | Troels T Nielsen | Q106557944 |
| P2093 | author name string | Jørgen E Nielsen | |
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| An RNA polymerase II construct synthesizes short-hairpin RNA with a quantitative indicator and mediates highly efficient RNAi | Q24806293 | ||
| Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice | Q27324749 | ||
| Pseudotype formation of murine leukemia virus with the G protein of vesicular stomatitis virus | Q27486172 | ||
| Prediction of mammalian microRNA targets | Q27860498 | ||
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| Asymmetry in the assembly of the RNAi enzyme complex | Q27860763 | ||
| 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 | ||
| The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14 | Q27860849 | ||
| Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans | Q27860867 | ||
| A system for stable expression of short interfering RNAs in mammalian cells | Q27860875 | ||
| Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells | Q27860926 | ||
| Dicer functions in RNA interference and in synthesis of small RNA involved in developmental timing in C. elegans | Q28131807 | ||
| LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 | Q28210584 | ||
| Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation | Q28246858 | ||
| Mice devoid of PrP are resistant to scrapie | Q28249108 | ||
| A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells | Q28249434 | ||
| Caspase cleavage of gene products associated with triplet expansion disorders generates truncated fragments containing the polyglutamine tract | Q28267110 | ||
| Second-generation shRNA libraries covering the mouse and human genomes | Q28275183 | ||
| Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 | Q28290118 | ||
| siRNA and miRNA: an insight into RISCs | Q28305768 | ||
| AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial | Q28307713 | ||
| In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector | Q29547503 | ||
| RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals | Q29547713 | ||
| An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells | Q29547714 | ||
| Recent advances with liposomes as pharmaceutical carriers | Q29616702 | ||
| The evolution of gene regulation by transcription factors and microRNAs | Q29617552 | ||
| A role for the RNase III enzyme DCR-1 in RNA interference and germ line development in Caenorhabditis elegans | Q29618304 | ||
| Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver | Q30434933 | ||
| RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells | Q30476196 | ||
| Regulation of neuropeptide mRNA expression in the basal ganglia by intrastriatal and intranigral transplants in the rat Parkinson model. | Q30924598 | ||
| Breeding of retroviruses by DNA shuffling for improved stability and processing yields | Q31832983 | ||
| A combinatorial library of lipid-like materials for delivery of RNAi therapeutics | Q33330928 | ||
| Alzheimer's-related endosome dysfunction in Down syndrome is Abeta-independent but requires APP and is reversed by BACE-1 inhibition. | Q33667620 | ||
| Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain | Q33731226 | ||
| Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates | Q33731243 | ||
| RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model | Q33936969 | ||
| A lentiviral microRNA-based system for single-copy polymerase II-regulated RNA interference in mammalian cells | Q33938488 | ||
| Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination | Q33938668 | ||
| Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi | Q33945342 | ||
| Expanding the microRNA targeting code: functional sites with centered pairing | Q34125341 | ||
| siRNA-mediated gene silencing in vitro and in vivo. | Q34150604 | ||
| RNA interference-mediated knockdown of alpha-synuclein protects human dopaminergic neuroblastoma cells from MPP(+) toxicity and reduces dopamine transport. | Q40203572 | ||
| Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7- and AAV-8-pseudotyped vectors | Q40238020 | ||
| Efficient transduction of neurons using Ross River glycoprotein-pseudotyped lentiviral vectors. | Q40309646 | ||
| Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi | Q40321534 | ||
| Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma | Q40365729 | ||
| Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model | Q40379114 | ||
| Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. | Q40397746 | ||
| Directed evolution of retrovirus envelope protein cytoplasmic tails guided by functional incorporation into lentivirus particles. | Q40478194 | ||
| Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. | Q40480552 | ||
| Overexpression of glial cell line-derived neurotrophic factor using a lentiviral vector induces time- and dose-dependent downregulation of tyrosine hydroxylase in the intact nigrostriatal dopamine system. | Q40532718 | ||
| Effects of RNA interference-mediated silencing of gamma-secretase complex components on cell sensitivity to caspase-3 activation | Q40547989 | ||
| Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease | Q40904090 | ||
| Production of high-titer human immunodeficiency virus type 1 pseudotyped with vesicular stomatitis virus glycoprotein | Q41097817 | ||
| Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice | Q41900569 | ||
| Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain | Q42280576 | ||
| Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response | Q42450986 | ||
| In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration | Q42502439 | ||
| Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients | Q42559350 | ||
| Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 | ||
| Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus | Q42798600 | ||
| Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats | Q42836546 | ||
| Efficient gene transfer into neurons in monkey brain by adeno-associated virus 8. | Q43132490 | ||
| Normal development and behaviour of mice lacking the neuronal cell-surface PrP protein | Q43522858 | ||
| Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model | Q43628011 | ||
| Selective modifications in GAD67 mRNA levels in striatonigral and striatopallidal pathways correlate to dopamine agonist priming in 6-hydroxydopamine-lesioned rats. | Q44658133 | ||
| Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic | Q44819303 | ||
| US gene therapy in crisis | Q44872256 | ||
| RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. | Q44964949 | ||
| Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs | Q45147309 | ||
| Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. | Q45225518 | ||
| Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. | Q45289792 | ||
| Cleavage at the caspase-6 site is required for neuronal dysfunction and degeneration due to mutant huntingtin | Q45301569 | ||
| Expression of mutated huntingtin fragment in the putamen is sufficient to produce abnormal movement in non-human primates | Q45305166 | ||
| Regional and progressive thinning of the cortical ribbon in Huntington's disease | Q45305508 | ||
| Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. | Q45377035 | ||
| Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain | Q45418748 | ||
| Herpes simplex virus RNAi and neprilysin gene transfer vectors reduce accumulation of Alzheimer's disease-related amyloid-beta peptide in vivo | Q45418795 | ||
| Stable suppression of tumorigenicity by virus-mediated RNA interference | Q45730805 | ||
| Quantitative analysis of the packaging capacity of recombinant adeno-associated virus | Q45766162 | ||
| Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector | Q45769447 | ||
| Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. | Q45871699 | ||
| No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. | Q45885410 | ||
| Increased exploratory activity of APP23 mice in a novel environment is reversed by siRNA. | Q46315786 | ||
| Efficient lentiviral vectors for short hairpin RNA delivery into human cells | Q46649798 | ||
| VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model | Q47427098 | ||
| Messenger RNAs encoding glutamate-decarboxylases are differentially affected by nigrostriatal lesions in subpopulations of striatal neurons | Q48505951 | ||
| Evidence for disease-regulated transgene expression in the brain with use of lentiviral vectors | Q48549527 | ||
| Lentivirus-mediated APP695-RNAi reduces apoptosis in APP transgenic mouse neurons | Q48859872 | ||
| Neuron-specific RNA interference using lentiviral vectors | Q39852828 | ||
| Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. | Q39913243 | ||
| Gene silencing in alveolar type II cells using cell-specific promoter in vitro and in vivo | Q40157495 | ||
| Directed evolution of novel adeno-associated viruses for therapeutic gene delivery. | Q34188892 | ||
| Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin | Q34221351 | ||
| Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis | Q34224830 | ||
| Oncoretrovirus and lentivirus vectors pseudotyped with lymphocytic choriomeningitis virus glycoprotein: generation, concentration, and broad host range | Q34329994 | ||
| AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method | Q34330377 | ||
| Advances with RNA interference in Alzheimer's disease research. | Q34330911 | ||
| In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River Virus glycoproteins | Q34344453 | ||
| Transcription and processing of human microRNA precursors | Q34378218 | ||
| Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism | Q34492631 | ||
| Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways | Q34530552 | ||
| Pathophysiology of Parkinson's disease: from clinical neurology to basic neuroscience and back | Q34596340 | ||
| Structure and expression of the Huntington's disease gene: evidence against simple inactivation due to an expanded CAG repeat | Q34662620 | ||
| Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi | Q34768688 | ||
| Introduction to retroviruses and retroviral vectors | Q35016620 | ||
| Pathophysiology of Parkinson's disease: the MPTP primate model of the human disorder. | Q35171033 | ||
| Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6. | Q35203559 | ||
| The influence of epileptic neuropathology and prior peripheral immunity on CNS transduction by rAAV2 and rAAV5. | Q35341751 | ||
| Human immunodeficiency virus type 1-derived lentivirus vectors pseudotyped with envelope glycoproteins derived from Ross River virus and Semliki Forest virus | Q35543170 | ||
| CNS-specific therapy for ongoing EAE by silencing IL-17 pathway in astrocytes | Q36085387 | ||
| Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits | Q36089160 | ||
| Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells | Q36288360 | ||
| Silencing of Parkinson's disease-associated genes with artificial mirtron mimics of miR-1224. | Q36341509 | ||
| Cell systems and the toxic mechanism(s) of alpha-synuclein | Q36440972 | ||
| Engineering a serum-resistant and thermostable vesicular stomatitis virus G glycoprotein for pseudotyping retroviral and lentiviral vectors | Q37075772 | ||
| Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector | Q37251114 | ||
| Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice | Q37254850 | ||
| Knocking down barriers: advances in siRNA delivery | Q37380671 | ||
| Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs | Q37395054 | ||
| Neuropathological background of phenotypical variability in frontotemporal dementia | Q37879961 | ||
| Adeno-associated virus (AAV) gene therapy for neurological disease | Q37998750 | ||
| siRNA against presenilin 1 (PS1) down regulates amyloid β42 production in IMR-32 cells. | Q39418271 | ||
| Knockdown of GAD67 protein levels normalizes neuronal activity in a rat model of Parkinson's disease | Q39568396 | ||
| Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3? | Q39724719 | ||
| Divalent metal transporter 1 is involved in amyloid precursor protein processing and Abeta generation | Q39812912 | ||
| P275 | copyright license | Creative Commons Attribution 4.0 International | Q20007257 |
| P6216 | copyright status | copyrighted | Q50423863 |
| P433 | issue | 3 | |
| P921 | main subject | gene silencing | Q1431332 |
| neurodegeneration | Q1755122 | ||
| P304 | page(s) | 457-484 | |
| P577 | publication date | 2013-09-10 | |
| P1433 | published in | Genes | Q5532699 |
| P1476 | title | Antisense gene silencing: therapy for neurodegenerative disorders? | |
| P478 | volume | 4 |
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