申请人:Prosensa Technologies B.V.
公开号:EP2505211A1
公开(公告)日:2012-10-03
The invention provides conjugates, comprising an organ, tissue or tumor cell homing molecule linked to a moiety. Such a moiety can be, for example, an oligonucleotide, small interfering RNA, gene, virus, protein, pharmaceutical or detectable agent. In addition the invention provides methods to diagnose or treat a pathology of the muscle or heart, by administrating to a subject having or suspected of having a pathology a molecule or conjugate that homes to, binds to and is taken up by the muscle cells or heart cells.
本发明提供了由器官、组织或肿瘤细胞归巢分子与分子连接而成的共轭物。例如,这种分子可以是寡核苷酸、小干扰 RNA、基因、病毒、蛋白质、药物或可检测剂。此外,本发明还提供了诊断或治疗肌肉或心脏病变的方法,即向患有或疑似患有病变的受试者施用一种分子或共轭物,该分子或共轭物与肌肉细胞或心脏细胞归巢、结合并被其吸收。