Article

Neuron-derived extracellular vesicles in plasma present a potential non-invasive biomarker for Huntingtin protein and RNA assessment in Huntington disease

2025-07-21

Abstract excerpt

Huntington disease (HD) is a neurodegenerative disease caused by a trinucleotide repeat expansion in the HTT gene encoding an elongated polyglutamine tract in the huntingtin (HTT) protein. The use of biomarkers has become a major component in preclinical studies focusing on HTT lowering strategies. Quantification of soluble mutant HTT (mHTT) in cerebrospinal fluid (CSF) has served as a pharmacodynamic readout and...

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Literature Corpus work: 423f1b03-c86d-5ac8-9226-b657f7bbe2d9
DOI: 10.1101/2025.07.17.665403

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