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, HTT-lowering therapies hold promise. Recombinant adeno-assoc...

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Gepubliceerd in:Brain Sci
Hoofdauteurs: Spronck, Elisabeth A., Vallès, Astrid, Lampen, Margit H., Montenegro-Miranda, Paula S., Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M., Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, de Haan, Martin
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: MDPI 2021
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7908995/
https://ncbi.nlm.nih.gov/pubmed/33498212
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/brainsci11020129
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