Tailed Mirtron Effectors For RNAi-Mediated Gene Silencing

摘要

The present invention relates to an isolated mirtron capable of binding a target nucleic acid, wherein the isolated mirtron comprises: a) a stem loop structure comprising a 5' splice site, b) a tail sequence comprising a 3' splice site and a polypyrimidine-comprising sequence, and c) a branch point sequence. Further, the present invention relates to vectors and pharmaceutical compositions comprising such mirtrons as well as methods of treating a disease comprising administering such pharmaceutical compositions.