Materials and methods for treatment of autosomal dominant retinitis pigmentosa
US10662425B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Nov 21, 2018 |
| Grant date | May 26, 2020 |
| Priority date | — |
| Expiry date | Nov 21, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/80
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present application provides materials and methods for treating a patient with autosomal dominant RP, both ex vivo and in vivo; materials and methods for editing a RHO gene in a human cell; and materials and methods for editing a P23H mutation in a RHO gene in a human cell. In addition, the present application provides one or more gRNAs or sgRNAs for editing a RHO gene; one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a P23H mutation in a RHO gene. The present application provides a therapeutic for treating a patient with autosomal dominant RP. The present application provides a kit for treating a patient with autosomal dominant RP. In addition, the present application provides a self-inactivating CRISPR-Cas system.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.