Materials and methods for treatment of autosomal dominant retinitis pigmentosa
US12247201B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Apr 2, 2020 |
| Grant date | Mar 11, 2025 |
| Priority date | — |
| Expiry date | Jan 11, 2044 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/34
- 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.