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

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

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

http://www.wikidata.org/entity/Q33374523

about

Machado-Joseph Disease: from first descriptions to new perspectives.Cell biology of spinocerebellar ataxia Mouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II.Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice Current prospects for RNA interference-based therapies Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal projection neurons Overexpression of the autophagic beclin-1 protein clears mutant ataxin-3 and alleviates Machado-Joseph disease Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype.Huntington's disease: silencing a brutal killer.Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models Oligonucleotide-based strategies to combat polyglutamine diseases.Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi.RNA interference mitigates motor and neuropathological deficits in a cerebellar mouse model of Machado-Joseph disease An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1 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.Ubiquitin-binding site 2 of ataxin-3 prevents its proteasomal degradation by interacting with Rad23 RNAi medicine for the brain: progresses and challenges.Consensus paper: management of degenerative cerebellar disorders.Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6.Toward understanding Machado-Joseph disease Allele-selective inhibition of trinucleotide repeat genes.Ataxin-3 phosphorylation decreases neuronal defects in spinocerebellar ataxia type 3 models Recent advances in RNA interference therapeutics for CNS diseases.Fibroblasts of Machado Joseph Disease patients reveal autophagy impairment.Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics.Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease ss-siRNAs allele selectively inhibit ataxin-3 expression: multiple mechanisms for an alternative gene silencing strategy.Disease-causing allele-specific silencing by RNA interference.Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs.Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs.Silencing of CDK5 as potential therapy for Alzheimer's disease.RNA therapy for polyglutamine neurodegenerative diseases.Spinocerebellar ataxia type 2: clinical presentation, molecular mechanisms, and therapeutic perspectives.Research progress in allele-specific expression and its regulatory mechanisms.Lentiviral vector-mediated RNA silencing in the central nervous system.Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies.

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

http://www.wikidata.org/entity/Q33374523

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Allele-specific RNA silencing ...... del of Machado-Joseph disease.

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Gwennaëlle Auregan

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Isabel Nascimento-Ferreira

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Noëlle Dufour

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Philippe Hantraye

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Raymonde Hassig

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Sandro Alves

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P2860

An arginine/lysine-rich motif is crucial for VCP/p97-mediated modulation of ataxin-3 fibrillogenesis.

Ataxin-3 interactions with rad23 and valosin-containing protein and its associations with ubiquitin chains and the proteasome are consistent with a role in ubiquitin-mediated proteolysis

Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method

CAG expansions in a novel gene for Machado-Joseph disease at chromosome 14q32.1

RNA toxicity is a component of ataxin-3 degeneration in Drosophila

Inactivation of the mouse Atxn3 (ataxin-3) gene increases protein ubiquitination

Rational siRNA design for RNA interference

Spinocerebellar ataxia type 3 phenotypically resembling parkinson disease in a black family.

Ancestral origins of the Machado-Joseph disease mutation: a worldwide haplotype study

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

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e3341

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10

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Luis Pereira De Almeida

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Maria C Pedroso de Lima

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23305592

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18841197

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