CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models

CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models

In polyglutamine (polyQ) diseases, including Huntington disease (HD) and spinocerebellar ataxia type 3 (SCA3), targeting the mutant CAG tract in mRNA could be a therapeutic strategy for lowering pathogenic protein. We explored the viability of this therapeutic strategy in vivo at the level of the re...

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Bibliographic Details
Main Authors: Magdalena Surdyka, Żaneta Kalinowska-Pośka, Anna Niewiadomska-Cimicka, Ewelina Jesion, Agnieszka Fiszer, Elisabeth Singer-Mikosch, Lorraine Fievet, Lukasz Przybyl, Nicholas S. Caron, Michael R. Hayden, Huu Phuc Nguyen, Yvon Trottier, Maciej Figiel
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
short hairpin RNA
shRNA
CAG repeats targeting
Huntington disease
spinocerebellar ataxia type 3
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125000502
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