发明申请
WO2014204723A1 ONCOGENIC MODELS BASED ON DELIVERY AND USE OF THE CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS
审中-公开
基本信息:
- 专利标题： ONCOGENIC MODELS BASED ON DELIVERY AND USE OF THE CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS
- 专利标题（中）：基于交付和使用CRISPR-CAS系统，矢量和组成的ONCGENIC模型
- 申请号：PCT/US2014041790 申请日：2014-06-10
- 公开(公告)号：WO2014204723A1 公开(公告)日：2014-12-24
- 发明人： ZHANG FENG , EBERT BENJAMIN LEVINE , HECKL DIRK
- 申请人： BROAD INST INC , MASSACHUSETTS INST TECHNOLOGY , BRIGHAM WOMEN S HOSPITAL INC , ZHANG FENG , EBERT BENJAMIN LEVINE , HECKL DIRK
- 专利权人： BROAD INST INC,MASSACHUSETTS INST TECHNOLOGY,BRIGHAM WOMEN S HOSPITAL INC,ZHANG FENG,EBERT BENJAMIN LEVINE,HECKL DIRK
- 当前专利权人： BROAD INST INC,MASSACHUSETTS INST TECHNOLOGY,BRIGHAM WOMEN S HOSPITAL INC,ZHANG FENG,EBERT BENJAMIN LEVINE,HECKL DIRK
- 优先权： US201361915397 2013-12-12; US201361836123 2013-06-17
- 主分类号： C12N15/63
- IPC分类号： C12N15/63 ; C12N9/22 ; C12N15/10 ; C40B40/08
摘要：
The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to enable genome engineering in an organism to recapitulate the genetic complexity of disease or a condition and further interrogate gene function.
摘要（中）：
本发明提供了用于操纵靶序列的序列和/或活性的系统，方法和组合物的递送，工程和优化。 提供作为递送站点的递送系统和组织或器官。 还提供了载体和载体系统，其中一些编码CRISPR复合物的一个或多个组分，以及用于设计和使用这些载体的方法。 还提供了在真核细胞中引导CRISPR复合物形成的方法，以使生物体中的基因组工程重现疾病的遗传复杂性或病症并进一步询问基因功能。