发明名称 |
PRMT5 INHIBITORS FOR THE TREATMENT OF CANCER WITH REDUCED MTAP ACTIVTY |
摘要 |
The present invention generally relates to therapeutic inhibition of protein arginine methyltransferase 5 (PRMT5). In particular, cell lines having MTAP loss and increased intracellular MTA concentrations show selective dependence on PRMT5. Thus, the invention also relates to methods of identifying and treating PRMT5-related diseases in subjects or tissues which have an MTAP deficiency, alone or in combination, with a second agent that reduces MTAP activity and/or increases intracellular MTA levels, and/or provides an MTA analogs to the cell or tissue. The invention also relates to the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas System and components thereof. More specifically, the present invention relates to the delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions in tumor cells ex vivo and/or in vivo. For example using methods disclosed herein, cells can be sensitized to PRMT5 inhibition. |
申请公布号 |
WO2016145150(A3) |
申请公布日期 |
2016.10.27 |
申请号 |
WO2016US21697 |
申请日期 |
2016.03.10 |
申请人 |
THE BROAD INSTITUTE INC.;DANA-FARBER CANCER INSTITUTE, INC. |
发明人 |
GARRAWAY, Levi, A.;KRYUKOV, Grigoriy;RUTH, Jason;WILSON, Frederick |
分类号 |
A61K45/06;A61K31/7076;A61K45/00;A61P35/00 |
主分类号 |
A61K45/06 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|