发明名称 |
CRISPR/CAS9-BASED TREATMENTS |
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. |
申请公布号 |
WO2017004616(A1) |
申请公布日期 |
2017.01.05 |
申请号 |
WO2016US40962 |
申请日期 |
2016.07.05 |
申请人 |
THE JOHNS HOPKINS UNIVERSITY |
发明人 |
JUN, Albert, S.;RANGANATHAN, Vinod;ZACK, Donald |
分类号 |
C12N15/00;A61K48/00;A61P27/02;C12N9/22;C12N15/113;C12N15/90 |
主分类号 |
C12N15/00 |
代理机构 |
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地址 |
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