CHEMICAL MOLECULES THAT INHIBIT THE SLICING MECHANISM FOR TREATING DISEASES RESULTING FROM SPLICING ANOMALIES
申请人:Tazi Jamal
公开号:US20110053975A1
公开(公告)日:2011-03-03
The present invention relates to a compound of one of the formulas I to XXI; a pharmaceutical composition comprising at least one such compound; and the use of at least one such compound in preparing a drug to treat, in a subject, a genetic disease resulting from at least one splicing anomaly.
本发明涉及公式I至XXI中的化合物之一;包含至少一种这种化合物的药物组合物;以及使用至少一种这种化合物来制备一种药物,用于治疗主体中由至少一个剪接异常导致的遗传疾病。