THE CHILDRENS MEDICAL CENTER CORPORATION;THE CHILDREN'S MEDICAL CENTER CORPORATION;DANA-FARBER CANCER INSTITUTE, INC.
发明人:
BRADNER, JAMES,QI, JUN,BUCKLEY, DENNIS,ZON, LEONARD I.,MACARI, ELIZABETH
申请号：
CA3030719
公开号：
CA3030719A1
申请日:
2017.07.13
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
The present invention relates to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to the use of novel classes of compounds, i.e. inhibitors of RSK (p90S6K)； inhibitors of p70S6K； and inhibitors of rps6, to treat ribosomal disorders and ribosomopathies. In some embodiments, the invention relates to the use of specific Chk2 inhibitors and to the use of specific phenothiazine derivatives to treat ribosomal disorders and ribosomopathies, e.g. DBA.
