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Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
- Source :
- Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
- Publication Year :
- 2022
- Publisher :
- Nature Portfolio, 2022.
-
Abstract
- Chemically modified siRNAs distinguish between mutant and normal huntingtin based on a single nucleotide difference and lower mutant huntingtin specifically in patient derived cells and in a mouse model of Huntington’s disease.
- Subjects :
- Science
Subjects
Details
- Language :
- English
- ISSN :
- 20411723
- Volume :
- 13
- Issue :
- 1
- Database :
- Directory of Open Access Journals
- Journal :
- Nature Communications
- Publication Type :
- Academic Journal
- Accession number :
- edsdoj.b3726cec35ca4048a177c94f5d18c4bd
- Document Type :
- article
- Full Text :
- https://doi.org/10.1038/s41467-022-33061-x