Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph Disease

14 Jun 2013

Abstract excerpt

Machado-Joseph disease (MJD) is a dominantly inherited ataxia caused by a polyglutamine-coding expansion in the ATXN3 gene. Suppressing expression of the toxic gene product represents a promising approach to therapy for MJD and other polyglutamine diseases. We performed an extended therapeutic trial of RNA interference (RNAi) targeting ATXN3 in a mouse model expressing the full human disease gene and...

Source

Identifiers

PMID: 23765441
DOI: 10.1038/mt.2013.144

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Community interpretation

Discuss the evidence

Ask a question, add context or challenge an interpretation. Community activity is separate from scientific evidence strength.

0

discussions

0

replies

Citations encouraged

Claims remain attributable.

No discussion yet

Be the first to add a careful, cited interpretation.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph DiseasePMID 23765441Read ↗

RNA Interference Mitigates Motor and Neuropathological Deficits in a Cerebellar Mouse Model of Machado-Joseph DiseaseSemantic proximity Read ↗

rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3Semantic proximity Read ↗

Unravelling Endogenous MicroRNA System Dysfunction as a New Pathophysiological Mechanism in Machado-Joseph Disease.Semantic proximity Read ↗

Gene Editing for <i>ATXN3</i> Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced ToxicitySemantic proximity Read ↗

Recent therapeutic prospects for Machado–Joseph diseaseSemantic proximity Read ↗

Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.Semantic proximity Read ↗

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

Machado Joseph disease: clinical and genetic aspects, and current treatmentSemantic proximity Read ↗

AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3Semantic proximity Read ↗

Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice.Semantic proximity Read ↗

Oligonucleotide therapeutics in neurodegenerative diseasesSemantic proximity Read ↗

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse ModelsSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.