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...

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Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2019
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Article
Accesso online: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
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