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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Dettagli Bibliografici
Pubblicato in:Nucleic Acids Res
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2020
Soggetti:
Chemical Biology and Nucleic Acid Chemistry
Accesso online: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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