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

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 chall...

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Veröffentlicht in:J Neurosci
Hauptverfasser: 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.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Society for Neuroscience 2021
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7842749/
https://ncbi.nlm.nih.gov/pubmed/33310753
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.1865-20.2020
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