Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.
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Machado-Joseph Disease: from first descriptions to new perspectives.Precision medicine in spinocerebellar ataxias: treatment based on common mechanisms of diseaseOligonucleotide Therapies: The Past and the PresentMouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II.Antisense oligonucleotides: treating neurodegeneration at the level of RNASilencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic miceAutomated design of hammerhead ribozymes and validation by targeting the PABPN1 gene transcript.An Exocyclic Methylene Group Acts As a Bioisostere of the 2′-Oxygen Atom in LNAA dynamic structural model of expanded RNA CAG repeats: a refined X-ray structure and computational investigations using molecular dynamics and umbrella sampling simulations.Epigenetic mechanisms of neurodegeneration in Huntington's diseaseAllele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patientsTargeting several CAG expansion diseases by a single antisense oligonucleotideApproaches to Validate and Manipulate RNA Targets with Small Molecules in CellsTherapeutic prospects for spinocerebellar ataxia type 2 and 3.Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease.The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain.Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype.Huntington's disease: silencing a brutal killer.Quantification assays for total and polyglutamine-expanded huntingtin proteins.Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAsEvaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse ModelsEffect of post-mortem delay on N-terminal huntingtin protein fragments in human control and Huntington disease brain lysates.Oligonucleotide-based strategies to combat polyglutamine diseases.Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs.Design of embedded chimeric peptide nucleic acids that efficiently enter and accurately reactivate gene expression in vivoAn evaluation of oligonucleotide-based therapeutic strategies for polyQ diseasesPotent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.Potent and selective inhibition of A-to-I RNA editing with 2'-O-methyl/locked nucleic acid-containing antisense oligoribonucleotides.Targeting of repeated sequences unique to a gene results in significant increases in antisense oligonucleotide potency.Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts.Optimizing anti-gene oligonucleotide 'Zorro-LNA' for improved strand invasion into duplex DNA.Antisense therapy in neurologyRole of inositol 1,4,5-trisphosphate receptors in pathogenesis of Huntington's disease and spinocerebellar ataxiasAntisense and antigene inhibition of gene expression by cell-permeable oligonucleotide-oligospermine conjugates.
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Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on 03 May 2009
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@en
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@nl
type
label
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@en
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@nl
prefLabel
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@en
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@nl
P2093
P2860
P356
P1433
P1476
Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.
@en
P2093
David R Corey
Ilya Bezprozvanny
Jacob C Schwartz
Keith T Gagnon
Khalil Arar
Masayuki Matsui
Sylvie Gabillet
P2860
P2888
P304
P356
10.1038/NBT.1539
P577
2009-05-03T00:00:00Z