Cyclodextrin-Based Nanoparticles for Delivery of Antisense Oligonucleotides Targeting Huntingtin

• Główni autorzy: Monique C. P. Mendonça (Autor), Yao Sun (Autor), Michael F. Cronin (Autor), Andrew J. Lindsay (Autor), John F. Cryan (Autor), Caitriona M. O'Driscoll (Autor)
• Format: Książka
• Wydane: MDPI AG, 2023-02-01T00:00:00Z.
• Hasła przedmiotowe: article
• Dostęp online: Connect to this object online.

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 oli...