Invention Grant
- Patent Title: Materials and methods for treatment of autosomal dominant cone-rod dystrophy
-
Application No.: US16899717Application Date: 2020-06-12
-
Publication No.: US10995328B2Publication Date: 2021-05-04
- Inventor: Ryo Takeuchi , Akiko Noma , Shuying He , Abraham Scaria
- Applicant: CRISPR THERAPEUTICS AG , BAYER HEALTHCARE LLC
- Applicant Address: CH Zug; US NJ Whippany
- Assignee: CRISPR THERAPEUTICS AG,BAYER HEALTHCARE LLC
- Current Assignee: CRISPR THERAPEUTICS AG,BAYER HEALTHCARE LLC
- Current Assignee Address: CH Zug; US NJ Whippany
- Agency: Marshall, Gerstein & Borun LLP
- Main IPC: C12N9/22
- IPC: C12N9/22 ; C12N15/11 ; C12N15/90 ; A61K38/00

Abstract:
The present application provides materials and methods for treating a patient with autosomal dominant CORD, both ex vivo and in vivo; materials and methods for editing a GUCY2D gene in a human cell; and materials and methods for editing a R838H, R838C, or R838S mutation in a GUCY2D gene in a human cell. The present application also provides one or more gRNAs or sgRNAs for editing a GUCY2D gene; one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene. The present application provides a therapeutic for treating a patient with autosomal dominant CORD. The present application also provides a kit for treating a patient with autosomal dominant CORD. In addition, the present application provides a self-inactivating CRISPR-Cas system.
Public/Granted literature
- US1298922A Breather-tube and cap therefor. Public/Granted day:1919-04-01
Information query