- Patent Title: Materials and methods for treatment of primary hyperoxaluria type 1 (PH1) and other alanine-glyoxylate aminotransferase (AGXT) gene related conditions or disorders
-
Application No.: US16487282Application Date: 2018-02-21
-
Publication No.: US11407997B2Publication Date: 2022-08-09
- Inventor: Ante Sven Lundberg , Samarth Kulkarni , Lawrence Klein , Hari Kumar Padmanabhan
- Applicant: CRISPR THERAPEUTICS AG
- Applicant Address: CH Zug
- Assignee: CRISPR THERAPEUTICS AG
- Current Assignee: CRISPR THERAPEUTICS AG
- Current Assignee Address: CH Zug
- Agency: Marshall, Gerstein & Borun LLP
- International Application: PCT/IB2018/051072 WO 20180221
- International Announcement: WO2018/154459 WO 20180830
- Main IPC: C07H21/04
- IPC: C07H21/04 ; C07H21/02 ; C12N15/113 ; C12N9/22 ; C12N15/90

Abstract:
The present application provides materials and methods for treating a patient with one or more conditions or disorders associated with AGXT, both ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Primary Hyperoxaluria Type 1 (PH1). The present application also provides materials and methods for editing an AGXT gene in a cell by genome editing. The present application also provides materials and methods for altering a contiguous genomic sequence of an AGXT gene in a cell. In addition, the present application provides one or more gRNAs for editing an AGXT gene. The present application also provides a therapeutic comprising at least one or more gRNAs for editing an AGXT gene. In addition, the present application provides a therapeutic for treating a patient with an AGXT related condition or disorder.
Public/Granted literature
Information query