AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

Huntington disease (HD) is a fatal neurodegenerative genetic disorder, thought to reflect a toxic gain of function in huntingtin (Htt) protein. Adeno-associated viral vector serotype 5 (AAV5)- microRNA targeting huntingtin (miHTT) is a HD gene-therapy candidate that efficiently lowers HTT using RNAi...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6849441/
https://ncbi.nlm.nih.gov/pubmed/31737741
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.09.010
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