申请人:RAYAN ANWAR
公开号:US20100284959A1
公开(公告)日:2010-11-11
The present invention provides specific double-stranded DNA/RNA binding compounds having a polymeric structure, which are in fact, triplex forming molecules capable of binding tightly and specifically to predetermined sequences in the major groove of double stranded nucleic acid molecules; as well as pharmaceutical compositions comprising thereof. The triplex forming molecules and the pharmaceutical compositions of the invention can be used for various therapeutic applications such as site-specific modulation of gene expression and targeting of DNA or RNA damage, as well as for diagnostic applications in vitro.
本发明提供了具有聚合结构的特定双链DNA / RNA结合化合物,实际上是三链形成分子,能够紧密而特异地结合到双链核酸分子的主沟槽中的预定序列;以及包括它们的制药组合物。本发明的三链形成分子和制药组合物可用于各种治疗应用,例如位点特异性基因表达调节和DNA或RNA损伤的靶向,以及体外诊断应用。