| 发明名称 |
METHODS AND COMPOSITIONS FOR TREATMENT OF A GENETIC CONDITION |
| 摘要 |
Methods and compositions for genetic alteration of cells are provided. |
| 申请公布号 |
US2015056705(A1) |
申请公布日期 |
2015.02.26 |
| 申请号 |
US201414278903 |
申请日期 |
2014.05.15 |
| 申请人 |
Sangamo BioSciences, Inc. |
发明人 |
Conway Anthony;Cost Gregory J.;Dekelver Russell;Rebar Edward J.;Reik Andreas;Urnov Fyodor;Wang Jianbin;Zhang H. Steve |
| 分类号 |
C12N15/90;C12N15/11 |
主分类号 |
C12N15/90 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method of modifying the expression of an endogenous gene in a cell, the method comprising the steps of: administering to the cell a first nucleic acid molecule comprising a single guide RNA that recognizes a target site in the endogenous gene and a second nucleic acid molecule that encodes a functional domain, wherein the functional domain associates with the single guide RNA on the target site, thereby modifying the expression of the endogenous gene. |
| 地址 |
Richmond CA US |
