Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

Huntington disease (HD) is a fatal neurodegenerative disease caused by a pathogenic expansion of a CAG repeat in the huntingtin (HTT) gene. There are no disease-modifying therapies for HD. Artificial microRNAs targeting HTT transcripts for degradation have shown preclinical promise and will soon ent...

詳細記述

保存先:
書誌詳細
出版年:Nucleic Acids Res
主要な著者: Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2020
主題:
Chemical Biology and Nucleic Acid Chemistry
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7145682/
https://ncbi.nlm.nih.gov/pubmed/31745548
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz976
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