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

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 (<i>HTT</i>) gene. AAV5-miHTT is an adeno-associated virus serotype 5-based vector expressing an engineered HTT-targeting microRNA (miHTT). Preclinical studies demonstrate...

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Bibliographic Details
Main Authors: Roberto D. V. S. Morais, Marina Sogorb-González, Citlali Bar, Nikki C. Timmer, M. Leontien Van der Bent, Morgane Wartel, Astrid Vallès
Format: Article
Language:English
Published: MDPI AG 2022-09-01
Series:Cells
Subjects:
adeno-associated virus
exosomes
extracellular vesicles
gene therapy
Huntington’s disease
intercellular communication
Online Access:https://www.mdpi.com/2073-4409/11/17/2748

Internet

https://www.mdpi.com/2073-4409/11/17/2748

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