Invention Grant
- Patent Title: Materials and methods for treatment of autosomal dominant retinitis pigmentosa
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Application No.: US16198361Application Date: 2018-11-21
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Publication No.: US10662425B2Publication Date: 2020-05-26
- Inventor: Albena Kantardzhieva , Akiko Noma , Abraham Scaria , Ryo Takeuchi
- 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: Nelson Mullins Riley & Scarborough LLP
- Agent Amy E. Mandragouras, Esq.; Ariana D. Harris
- Main IPC: C12N15/11
- IPC: C12N15/11 ; A61K31/7088 ; A61K38/46 ; C12N9/22 ; C12N15/90 ; A61P27/02 ; C12N15/113

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.
Public/Granted literature
- US20190153441A1 MATERIALS AND METHODS FOR TREATMENT OF AUTOSOMAL DOMINANT RETINITIS PIGMENTOSA Public/Granted day:2019-05-23
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