Selective silencing of expanded CAG repeats: Chemically modified siRNA advances RNA-based therapies for polyglutamine diseases
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Authors
- Ronald A.M. Buijsen
- Gijs-Jan Scholten
- Jamie T. Hyde
Institutions
- Leiden University Medical Center (NL)
Publication Details
- Journal
- Molecular Therapy — Nucleic Acids
- Published
- 2026-02-25
- DOI
- https://doi.org/10.1016/j.omtn.2026.102869
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Hersenstichting
- HORIZON EUROPE Framework Programme