Invention Grant
- Patent Title: Rare-cutting endonucleases for efficient and specific targeting DNA sequences comprising highly repetitive motives
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Application No.: US15031996Application Date: 2014-10-24
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Publication No.: US10508289B2Publication Date: 2019-12-17
- Inventor: Philippe Duchateau , Alexandre Juillerat
- Applicant: CELLECTIS, S.A.
- Applicant Address: FR Paris
- Assignee: CELLECTIS
- Current Assignee: CELLECTIS
- Current Assignee Address: FR Paris
- Agency: Arrigo, Lee, Guttman & Mouta-Bellum LLP
- Priority: DK201370623 20131025
- International Application: PCT/EP2014/072833 WO 20141024
- International Announcement: WO2015/059265 WO 20150430
- Main IPC: C12N15/90
- IPC: C12N15/90 ; C12N9/22 ; A61K48/00 ; C07K14/195 ; A61K38/00

Abstract:
The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.
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