Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. - Wikidata - wikimedia - TriplyDB

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.

http://www.wikidata.org/entity/Q37395054

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Machado-Joseph Disease: from first descriptions to new perspectives.Precision medicine in spinocerebellar ataxias: treatment based on common mechanisms of disease Oligonucleotide Therapies: The Past and the Present Mouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II.Antisense oligonucleotides: treating neurodegeneration at the level of RNA Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice Automated 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 LNA A 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 disease Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients Targeting several CAG expansion diseases by a single antisense oligonucleotide Approaches to Validate and Manipulate RNA Targets with Small Molecules in Cells Therapeutic 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 RNAs Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models Effect 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 vivo An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases Potent 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 1 Sustained 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 neurology Role of inositol 1,4,5-trisphosphate receptors in pathogenesis of Huntington's disease and spinocerebellar ataxias Antisense 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.

http://www.wikidata.org/entity/Q37395054

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article científic

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article scientifique

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articolo scientifico

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artigo científico

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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.

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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

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Nature Biotechnology

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Allele-specific silencing of m ...... expanded CAG repeats in mRNAs.

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David R Corey

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Ilya Bezprozvanny

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Jacob C Schwartz

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Jiaxin Hu

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Jun Wu

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Keith T Gagnon

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Khalil Arar

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Masayuki Matsui

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Sylvie Gabillet

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Huntington's disease

Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes

Huntingtin is required for neurogenesis and is not impaired by the Huntington's disease CAG expansion

Trinucleotide Repeat Disorders

Designing siRNA that distinguish between genes that differ by a single nucleotide

Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease.

Disturbed Ca2+ signaling and apoptosis of medium spiny neurons in Huntington's disease.

RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.

RNA structure of trinucleotide repeats associated with human neurological diseases.

Antisense properties of duplex- and triplex-forming PNAs.

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478-484

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scholarly article

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10.1038/NBT.1539

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5

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27

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2009-05-03T00:00:00Z

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19412185

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