Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism. There are currently no disease modifying therapies for HD; however, <i>HTT</i>-lowering therapies hold promise. Reco...

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Bibliographic Details
Main Authors: Elisabeth A. Spronck, Astrid Vallès, Margit H. Lampen, Paula S. Montenegro-Miranda, Sonay Keskin, Liesbeth Heijink, Melvin M. Evers, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Martin de Haan
Format: Article
Language:English
Published: MDPI AG 2021-01-01
Series:Brain Sciences
Subjects:
AAV5
Huntington disease
miRNA
gene therapy
Online Access:https://www.mdpi.com/2076-3425/11/2/129

Internet

https://www.mdpi.com/2076-3425/11/2/129

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