Specific synthetic chimeric Xenonucleic acid guide RNA; s(XNA-gRNA) for enhancing CRISPR mediated genome editing efficiency
US11274302B2 · kind B2 · utility
Assignee
Inventor
Key dates
| Filing date | Oct 17, 2017 |
| Grant date | Mar 15, 2022 |
| Priority date | — |
| Expiry date | Apr 8, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/3519
- WIPO fieldBiotechnology
- WIPO sectorChemistry
Abstract
The invention provides specific synthetic chimeric xenonucleic acid guide RNA; s(XNA-gRNA) for enhancing crispr mediated genome editing efficiency. The invention also provides methods and compositions for inducing CRISPR/Cas-based gene editing/regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using single guide RNAs (sgRNAs) that have been chemically modified with xeno nucleic acids which enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a sgRNA that has been chemically modified with xeno nucleic acids to correct a mutation in a target gene associated with the genetic disease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.