Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
US10577630B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Dec 16, 2015 |
| Grant date | Mar 3, 2020 |
| Priority date | — |
| Expiry date | Dec 16, 2035 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y301/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.