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Antagomirs and RNA sponges are both agents that are used to silence microRNAs. Antagomirs are chemically modified oligonucleotides that bind specifically to particular microRNAs and sponge RNAs are small synthetic RNAs that bind to multiple microRNAs that have the same sequence in their ‘seed region’.
Engineered RNA endonuclease XNAzymes can specifically target individual disease-associated non-coding RNAs (ncRNAs) in vitro with potential as platform technology for precision detection or knockdown of individual microRNAs or longer ncRNAs.
Depletion of the splicing factors MBNL 1 and 2 causes myotonic dystrophy. Here, the authors show that miR-23b and miR-218 target MBNL proteins, and that antagonists to these miRNAs rescue mis-splicing events in myoblasts and boost MBNL expression and rescue pathology in mouse models.
MicroRNAs regulate a wide range of biological processes and being able to inhibit their function could allow the development of therapeutic options. Here the authors describe a ‘small RNA zipper’ that sequesters miRNAs by forming a chain of DNA:RNA duplexes.
The findings from a preliminary report on a Phase I clinical trial of a targeted small interfering RNA drug and the use of an antagomir to target a microRNA associated with metastasis are discussed.