Targeting of microRNAs for therapeutics.
miRNAs (microRNAs) comprise a class of small endogenous non-coding RNAs that post-transcriptionally repress gene expression by base-pairing with their target mRNAs. Recent evidence has shown that miRNAs play important roles in a wide variety of human diseases, such as viral infections, cancer and cardiovascular diseases, and thus miRNAs have rapidly emerged as potential targets for therapeutics. LNAs (locked nucleic acids) comprise a class of bicyclic conformational analogues of RNA, which exhibit high binding affinity to complementary RNA molecules and high stability in blood and tissues in vivo. Recent reports on LNA-mediated miRNA silencing in rodents and primates support the potential of LNA-modified oligonucleotides in studying miRNA functions in vivo and in the future development of miRNA-based therapeutics.
Stenvang
Jan
J
Santaris Pharma, Hørsholm, Denmark.
Lindow
Morten
M
Kauppinen
Sakari
S
D000818:Animals; D004194:Disease; D006801:Humans; D051379:Mice; D035683:MicroRNAs / Q000037:antagonists & inhibitors / Q000627:therapeutic use*; D009841:Oligonucleotides / Q000737:chemistry / Q000494:pharmacology; D011323:Primates
D035683:MicroRNAs; D009841:Oligonucleotides; C477371:locked nucleic acid