Regneration Project mRNA
Messenger Ribonucleic acid Therapeutics (mRNA-T)
RNA interference (RNAi) is being widely used in functional gene research and is an important tool for drug discovery. However, canonical double-stranded short interfering RNAs are unstable and induce undesirable adverse effects, and thus there is no currently RNAi-based therapy in the clinic. Scientists have developed a novel class of RNAi agents, and evaluated their effectiveness in vitro and in mouse models. The novel class of RNA were synthesized on solid phase as single-stranded RNAs that, following synthesis, self-anneal into a unique helical structure containing a central stem and two loops. They are resistant to degradation and suppress their target genes. Agents directed against TGF ameliorate outcomes and induce no off-target effects in three animal models of lung disease. The results of this study support the pathological relevance of TGF in lung diseases, and suggest the potential usefulness of these novel RNA agents for therapeutic application.
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