CHEMICAL MOLECULES THAT INHIBIT THE SLICING MECHANISM FOR TREATING DISEASES RESULTING FROM SPLICING ANOMALIES
申请人:CENTRE NATIONAL DE RECHERCHE SCIENTIFIQUE
公开号:US20140155436A1
公开(公告)日:2014-06-05
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到XXI中的化合物,包括至少一种这样的化合物的制药组合物,以及使用至少一种这样的化合物制备药物,用于治疗由至少一种剪接异常导致的遗传疾病的受体。