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...

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Bibliografske podrobnosti
izdano v:Nucleic Acids Res
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2020
Teme:
Chemical Biology and Nucleic Acid Chemistry
Online dostop: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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