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Rare-cutting endonucleases for efficient and specific targeting DNA sequences comprising highly repetitive motives
专利权人:
CELLECTIS; S.A.
发明人:
Philippe Duchateau,Alexandre Juillerat
申请号:
US15031996
公开号:
US10508289B2
申请日:
2014.10.24
申请国别(地区):
US
年份:
2019
代理人:
摘要:
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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