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CRISPR/Cas9-based treatments
专利权人:
The Johns Hopkins University
发明人:
Jun, Albert S.,Jaskula-Ranga, Vinod,Zack, Donald
申请号:
AU2016287836
公开号:
AU2016287836A1
申请日:
2016.07.05
申请国别(地区):
AU
年份:
2018
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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