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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Détails bibliographiques
Publié dans:Mol Ther Methods Clin Dev
Auteurs principaux: Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Format: Artigo
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2019
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Article
Accès en ligne: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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