Cyclodextrin-Based Nanoparticles for Delivery of Antisense Oligonucleotides Targeting Huntingtin

Huntington’s disease (HD) is a progressive inherited neurodegenerative disease caused by a CAG repeat expansion in the huntingtin gene, which is translated into the pathologic mutant huntingtin (mHTT) protein. Despite the great potential of HTT lowering strategies and the numerous antisense oligonuc...

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Bibliographic Details
Main Authors: Monique C. P. Mendonça, Yao Sun, Michael F. Cronin, Andrew J. Lindsay, John F. Cryan, Caitriona M. O’Driscoll
Format: Article
Language:English
Published: MDPI AG 2023-02-01
Series:Pharmaceutics
Subjects:
Online Access:https://www.mdpi.com/1999-4923/15/2/520