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Article

Mutant Huntingtin Is Cleared from the Brain via Active Mechanisms in Huntington Disease.

The Journal of neuroscience : the official journal of the Society for Neuroscience - 27 Jan 2021

Caron Nicholas S, Banos Raul, Yanick Christopher, Aly Amirah E, Byrne Lauren M, Smith Ethan D, Xie Yuanyun, Smith Stephen E P, Potluri Nalini, Findlay Black Hailey, Casal Lorenzo, Ko Seunghyun, Cheung Daphne, Kim Hyeongju, Seong Ihn Sik, Wild Edward J, Song Ji-Joon, Hayden Michael R, Southwell Amber L

Abstract excerpt

Huntington disease (HD) is a neurodegenerative disease caused by a CAG trinucleotide repeat expansion in the huntingtin (HTT) gene. Therapeutics that lower HTT have shown preclinical promise and are being evaluated in clinical trials. However, clinical assessment of brain HTT lowering presents challenges. We have reported that mutant HTT (mHTT) in the CSF of HD patients correlates with clinical measures,...

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Identifiers

PMID: 33310753
DOI: 10.1523/JNEUROSCI.1865-20.2020
PMCID: PMC7842749

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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