Invention Application
- Patent Title: THERAPEUTIC AGENT FOR DISEASE CAUSED BY DOMINANT MUTANT GENE
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Application No.: US17626042Application Date: 2020-07-10
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Publication No.: US20220249698A1Publication Date: 2022-08-11
- Inventor: Masayo TAKAHASHI , Akishi ONISHI , Yuji TSUNEKAWA
- Applicant: RIKEN
- Applicant Address: JP Saitama
- Assignee: RIKEN
- Current Assignee: RIKEN
- Current Assignee Address: JP Saitama
- Priority: JP2019-130199 20190712
- International Application: PCT/JP2020/026956 WO 20200710
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K38/46 ; A61K31/7088 ; A61K38/17 ; A61P27/02

Abstract:
Provided is a novel therapeutic agent for a disease caused by a dominant gene mutation (7). A therapeutic agent of the present invention comprises a donor DNA (20) that contains a polynucleotide having the following sequences (a) to (c): (a) a normal gene (1); (b) a first reverse target sequence (2a) that is located upstream of the normal gene (1) and that is cleaved by a designer nuclease; and (c) a second reverse target sequence (2b) that is located downstream of the normal gene (1) and that is cleaved by the designer nuclease, where the reverse target sequences (2a, 2b) each mean a sequence obtained by inverting a target sequence (6) that is present in the genome and that is cleaved by the designer nuclease.
Information query