公开(公告)号：EP3230451A1

公开(公告)日：2017-10-18

申请号：EP15819996.8

申请日：2015-12-11

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  President and Fellows of Harvard College

发明人： ZHANG, Feng ,  ABUDAYYEH, Omar, O. ,  DAHLMAN, James, E. ,  HSU, Patrick ,  SCOTT, David, A.

IPC分类号： C12N15/11

CPC分类号： C12N15/907 ,  A01K67/0275 ,  A01K2217/07 ,  A01K2227/105 ,  C07K14/4702 ,  C12N9/22 ,  C12N9/96 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/20 ,  C12Y301/21004

摘要： The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems, wherein the guide sequence is modified by secondary structure to increase the specificity of the CRISPR-Cas system and whereby the secondary structure can protect against exonuclease activity and allow for 5′ additions to the guide sequence.

摘要翻译： 本发明提供了用于改变靶基因序列和相关基因产物表达的系统，方法和组合物。 提供了关于CRISPR-Cas系统的Cas蛋白的结构信息，使用该信息产生CRISPR复合物的修饰组分，编码CRISPR复合物的一种或多种组分或修饰组分的载体和载体系统，以及方法 用于这些载体和组件的设计和使用。 还提供了指导真核细胞中CRISPR复合物形成的方法和利用CRISPR-Cas系统的方法。 特别地，本发明包括优化的功能性CRISPR-Cas酶系统，其中指导序列通过二级结构修饰以增加CRISPR-Cas系统的特异性，并且其中二级结构可以保护免受外切核酸酶活性，并允许5'加入 指导顺序。

公开(公告)号：EP2691443A2

公开(公告)日：2014-02-05

申请号：EP12724782.3

申请日：2012-03-23

申请人： Massachusetts Institute Of Technology

发明人： DAHLMAN, James, E. ,  SCHROEDER, Avraham, D. ,  ANDERSON, Daniel, Griffith ,  LANGER, Robert, S. ,  LEVINS, Christopher, G.

IPC分类号： C08G73/10

CPC分类号： A61K47/22 ,  A61K9/0019 ,  A61K9/1272 ,  A61K47/34 ,  A61K47/543 ,  A61K47/59 ,  A61K47/6907 ,  A61K47/6911 ,  A61K47/6921 ,  C07D257/02 ,  C07D273/00 ,  C07D273/08 ,  C07D295/084 ,  C08G73/10 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2320/32

摘要： The present invention provides inventive conjugated polyethyleneimine (PEI) polymers and conjugated aza-macrocycles (collectively referred to herein as “conjugated lipomers” or “lipomers”) containing one or more groups of the formula (iii): wherein R3 and R4 are as defined herein. Also provided are compositions comprising the inventive conjugated lipomers, and methods of preparation and use.