Article

Challenges and advances for huntingtin detection in cerebrospinal fluid: in support of relative quantification

2024-09-27

Abstract excerpt

Huntington disease (HD) is a progressive and devastating neurodegenerative disease caused by expansion of a glutamine-coding CAG tract in the huntingtin ( HTT ) gene above a critical threshold of ∼35 repeats resulting in expression of mutant HTT (mHTT). A promising treatment approach being tested in clinical trials is HTT lowering, which aims to reduce levels of the mHTT protein. Target engagement of these therap...

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Literature Corpus work: 62f4cfa1-ed15-5d94-9048-10d84f5aaf05
DOI: 10.1101/2024.09.25.614766

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