THE CHILDREN'S MEDICAL CENTER CORPORATION;DANA-FARBER CANCER INSTITUTE, INC.
发明人:
James BRADNER,Jun QI,Dennis BUCKLEY,Leonard I. ZON,Elizabeth MACARI
申请号：
US16315899
公开号：
US20190314374A1
申请日:
2017.07.13
申请国别(地区):
US
年份:
2019
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.
