公开(公告)号：US20210071181A1

公开(公告)日：2021-03-11

申请号：US17061534

申请日：2020-10-01

申请人： ROCHE INNOVATION CENTER COPENHAGEN A/S

发明人： Joacim ELMEN ,  Phil KEARNEY ,  Sakari KAUPPINEN

IPC分类号： C12N15/113

摘要： The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 17 nucleobases which are complementary to human microRNAs. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogues into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the mIRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC.