The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).
本发明一般涉及用于治疗
核糖体紊乱和
核糖体病(如菱形黑帆贫血症(
DBA))的方法、组合物和试剂盒。在某些实施方案中,本发明涉及使用
钙调蛋白
抑制剂和
钙通道阻滞剂治疗
核糖体紊乱和
核糖体病(如钻石黑范贫血症(
DBA))的方法。