Back to search

Article

Polyglutamine-expanded ataxin-3: a target engagement marker for Spinocerebellar ataxia type 3 in peripheral blood

2021-04-05

Abstract excerpt

<title>Abstract</title> <p>Spinocerebellar ataxia type 3 is a rare neurodegenerative disease, caused by a CAG repeat expansion leading to polyglutamine elongation in the ataxin-3 protein. While no curative therapy is yet available, preclinical gene silencing approaches to reduce polyglutamine-toxicity demonstrate promising results. In view of upcoming clinical trials, quantitative and easily accessible molecular...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
b1509005-d57d-525e-ac29-e15adcadf054
DOI
10.21203/rs.3.rs-384325/v1
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
Polyglutamine-expanded ataxin-3: a target engagement marker for Spinocerebellar ataxia type 3 in peripheral bloodDOI 10.21203/rs.3.rs-384325/v1
Polyglutamine‐Expanded Ataxin‐3: A Target Engagement Marker for Spinocerebellar Ataxia Type 3 in Peripheral BloodSemantic proximity
Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3.Semantic proximity
Development of a highly sensitive immunoassay to measure ataxin2 as a target engagement marker in Spinocerebellar Ataxia Type 2Semantic proximity
Mutant Ataxin-3-Containing Aggregates (MATAGGs) in Spinocerebellar Ataxia Type 3: Dynamics of the Disorder.Semantic proximity
Cerebellar soluble mutant ataxin-3 level decreases during disease progression in Spinocerebellar Ataxia Type 3 mice.Semantic proximity
Small non-coding RNA content in plasma-derived extracellular vesicles distinguish ataxic SCA3 mutation carriers from pre-ataxic and control subjectsSemantic proximity
Identification of a Potential Exosomal Biomarker in Spinocerebellar Ataxia Type 3/Machado–Joseph DiseaseSemantic proximity
Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon.Semantic proximity
Therapeutic effects of engineered exosome-based miR-25 and miR-181a treatment in spinocerebellar ataxia type 3 mice by silencing ATXN3Semantic proximity
Comprehensive preclinical evaluation of human-derived anti-poly-GA antibodies in cellular and animal models of C9ORF72 diseaseSemantic proximity
Flow cytometry allows rapid detection of protein aggregates in cell culture and zebrafish models of spinocerebellar ataxia-3Semantic proximity
AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3Semantic proximity
Select a neighboring publication to make it the new centre.