Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease - Wikidata - awgldk - TriplyDB

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

http://www.wikidata.org/entity/Q37254846

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Precision medicine in spinocerebellar ataxias: treatment based on common mechanisms of disease Serotonergic signalling suppresses ataxin 3 aggregation and neurotoxicity in animal models of Machado-Joseph disease.Oligonucleotide-based strategies to combat polyglutamine diseases.RNA interference mitigates motor and neuropathological deficits in a cerebellar mouse model of Machado-Joseph disease Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Ubiquitin-binding site 2 of ataxin-3 prevents its proteasomal degradation by interacting with Rad23 Nonallele specific silencing of ataxin-7 improves disease phenotypes in a mouse model of SCA7.Population genetics and new insight into range of CAG repeats of spinocerebellar ataxia type 3 in the Han Chinese population.Differential recruitment of UBQLN2 to nuclear inclusions in the polyglutamine diseases HD and SCA3.Cellular miR-130b inhibits replication of porcine reproductive and respiratory syndrome virus in vitro and in vivo.Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice RNA viruses and microRNAs: challenging discoveries for the 21st century.Discovery of Therapeutic Approaches for Polyglutamine Diseases: A Summary of Recent Efforts.Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain.Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7.Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice Unbiased screen identifies aripiprazole as a modulator of abundance of the polyglutamine disease protein, ataxin-3.Polyglutamine spinocerebellar ataxias - from genes to potential treatments.Epigenetic profiles in polyglutamine disorders.Neurochemical abnormalities in premanifest and early spinocerebellar ataxias.Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model.

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P2860

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

http://www.wikidata.org/entity/Q37254846

description

article científic

@ca

article scientifique

@fr

articolo scientifico

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artigo científico

@pt

bilimsel makale

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scientific article published on 14 June 2013

@en

vedecký článok

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vetenskaplig artikel

@sv

videnskabelig artikel

@da

vědecký článek

@cs

name

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

@en

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease.

@nl

type

label

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

@en

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease.

@nl

prefLabel

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

@en

Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease.

@nl

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Molecular Therapy

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Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease

@en

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Beverly L Davidson

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Edgardo Rodríguez-Lebrón

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Henry L Paulson

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Katiuska Luna-Cancalon

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Lucas Martin-Fishman

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Maria do Carmo Costa

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Michelle Ouyang

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Naila S Ashraf

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Rahil M Dharia

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Svetlana Fischer

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P2860

Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

Silencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic mice

Improvement in the molecular diagnosis of Machado-Joseph disease

The genomic structure and expression of MJD, the Machado-Joseph disease gene

Machado-Joseph disease gene products carrying different carboxyl termini

Population genetics of wild-type CAG repeats in the Machado-Joseph disease gene in Portugal

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

Lentivector-mediated rescue from cerebellar ataxia in a mouse model of spinocerebellar ataxia.

Neuron-specific proteotoxicity of mutant ataxin-3 in C. elegans: rescue by the DAF-16 and HSF-1 pathways

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

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1898-1908

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scholarly article

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10.1038/MT.2013.144

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10

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2013-06-14T00:00:00Z

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23765441

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3808129

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