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TRAITEMENTS A BASE DE CRISPR/CAS9
专利权人:
THE JOHNS HOPKINS UNIVERSITY
发明人:
JUN, ALBERT, S.,JASKULA-RANGA, VINOD,ZACK, DONALD
申请号:
CA2989331
公开号:
CA2989331A1
申请日:
2016.07.05
申请国别(地区):
CA
年份:
2017
代理人:
摘要:
Described herein are methods for treating disorders affecting ocular and non-ocular tissue, such as corneal dystrophies and microsatellite expansion diseases. The methods use a nuclease system, such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR associated (Cas) 9 (CRISPR-Cas9), to cut and/or repair genomic DNA. Such methods may further comprise a DNA double-stranded break (DSB) repair system comprising a repair template in combination with a Non-Homologous End- Joining (NHEJ) or Homology Directed Repair (HDR) targeted to the one or more CRISPR-Cas9 cleavage sites.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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