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Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study

Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by GAG expansion in exon 1 of the huntingtin (HTT) gene. AAV5-miHTT is an adeno-associated virus serotype 5-based vector expressing an engineered HTT-targeting microRNA (miHTT). Preclinical studies demonstrate the brain-wide spre...

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Detalles Bibliográficos
Autores principales:	Morais, Roberto D. V. S., Sogorb-González, Marina, Bar, Citlali, Timmer, Nikki C., Van der Bent, M. Leontien, Wartel, Morgane, Vallès, Astrid
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9455173/ https://www.ncbi.nlm.nih.gov/pubmed/36078156 http://dx.doi.org/10.3390/cells11172748

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