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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Auteurs principaux:	Sonay Keskin, Cynthia C. Brouwers, Marina Sogorb-Gonzalez, Raygene Martier, Josse A. Depla, Astrid Vallès, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Format:	Artigo
Langue:	Inglês
Publié:	Elsevier 2019-12-01
Collection:	Molecular Therapy: Methods & Clinical Development
Accès en ligne:	http://www.sciencedirect.com/science/article/pii/S2329050119301081
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