Allele selective gene editing and uses thereof
US10369232B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Sep 21, 2016 |
| Grant date | Aug 6, 2019 |
| Priority date | — |
| Expiry date | Sep 21, 2036 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/34
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
This invention encompasses compounds, structures, compositions and methods for therapeutic guide molecules that direct CRISPR gene editing. A guide molecule for directing gene editing can be allele selective, or disease allele selective, and can exhibit reduced off target activity. A guide molecule can be composed of monomers, including UNA monomers, nucleic acid monomers, and modified nucleotides, wherein the compound is targeted to a genomic DNA. The guide molecules of this invention can be used as active ingredients for editing or disrupting a gene in vitro, ex vivo, or in vivo.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.