AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

Huntington disease (HD) is a fatal neurodegenerative disorder caused by an autosomal dominant CAG repeat expansion in the huntingtin (HTT) gene. The translated expanded polyglutamine repeat in the HTT protein is known to cause toxic gain of function. We showed previously that strong HTT lowering pre...

詳細記述

保存先:
書誌詳細
出版年:Mol Ther Methods Clin Dev
主要な著者: Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Gene & Cell Therapy 2019
主題:
Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6446047/
https://ncbi.nlm.nih.gov/pubmed/30984798
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.03.002
タグ: タグ追加
タグなし, このレコードへの初めてのタグを付けませんか!

類似資料