RNA-based Therapeutics- Current Progress and Future Prospects
By John C. Burnett, John J. Rossi. Chemistry and Biology, 2012
879 citations recorded by the source corpus.
Summary Recent advances of biological drugs have broadened the scope of therapeutic targets for a variety of human diseases. This holds true for dozens of RNA-based therapeutics currently under clinical investigation for diseases ranging from genetic disorders to HIV infection to various cancers. These emerging drugs, which include therapeutic ribozymes, aptamers, and small interfering RNAs (siRNAs), demonstrate the unprecedented versatility of RNA. However, RNA is inherently unstable, potentially immunogenic, and typically requires a delivery vehicle for efficient transport to the targeted cells. These issues have hindered the clinical progress of some RNA-based drugs and have contributed to mixed results in clinical testing. Nevertheless, promising results from recent clinical trials suggest that these barriers may be overcome with improved synthetic delivery carriers and chemical modifications of the RNA therapeutics. This review focuses on the clinical results of siRNA, RNA aptamer, and ribozyme therapeutics and the prospects for future successes.
Subfields: Molecular Biology
View Paper: https://www.semanticscholar.org/paper/865c63007b22df5f27c4eb86c7cfb124e1704554
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In Molecular Biology
Citations in the corpus
- Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine (2017, background): “This drug is being developed to prevent acute kidney injury (AKI) following primary cardiovascular surgery, as well as for prophylaxis of the delayed graft function (DGF) after deceased donor renal transplantation.(34) It has been noted that”
- The delivery of therapeutic oligonucleotides (2016, background): “Because of their selectivity, siRNAs have seen widespread use in the laboratory and there is great interest in their potential therapeutic applications (26).”