发明公开
EP3080257A1 COMPOSITIONS AND METHODS OF USE OF CRISPR-CAS SYSTEMS IN NUCLEOTIDE REPEAT DISORDERS
审中-公开
基本信息:
- 专利标题： COMPOSITIONS AND METHODS OF USE OF CRISPR-CAS SYSTEMS IN NUCLEOTIDE REPEAT DISORDERS
- 专利标题（中）：组合物及其使用方法CRISPR-CAS系统核苷酸重复病症
- 申请号：EP14821429.9 申请日：2014-12-12
- 公开(公告)号：EP3080257A1 公开(公告)日：2016-10-19
- 发明人： ZHANG, Feng , DAVIDSON, Beverly , LIN, Chie-yu , RODRIGUEZ, Edgardo
- 申请人： The Broad Institute Inc. , Massachusetts Institute of Technology , University of Iowa Research Foundation
- 申请人地址： 415 Main Street Cambridge, MA 02142 US
- 专利权人： The Broad Institute Inc.,Massachusetts Institute of Technology,University of Iowa Research Foundation
- 当前专利权人： The Broad Institute Inc.,Massachusetts Institute of Technology,University of Iowa Research Foundation
- 当前专利权人地址： 415 Main Street Cambridge, MA 02142 US
- 代理机构： V.O.
- 优先权： US201361915150P 20131212; US201462010879P 20140611; US201462010888P 20140611
- 国际公布： WO2015089351 20150618
- 主分类号： C12N9/22
- IPC分类号： C12N9/22 ; C12N15/10 ; C12N15/85
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 especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
摘要（中）：
本发明提供了系统，方法，和组合物的序列和/或靶序列的活动的操作输送，工程和优化爱尤其用作核苷酸重复障碍。 提供了哪些有针对性的网站进行传递真爱特别是用作核苷酸重复障碍输送系统和组织或器官。 因此，提供了用于设计和追求的载体和载体系统其中的一些编码复杂或CRISPR系统的爱情一个或多个组件特别是用作核苷酸重复障碍，以及方法。 所以提供了在真核细胞中指导CRISPR复合物或系统形成方法特别爱用作到核苷酸重复障碍考虑特异性目标识别和毒性和编辑的避免或在感兴趣的基因组基因座修饰的靶位点，以改变或包括 提高疾病或症状的状态。