Emerging developments in RNA therapeutics.
Exciting developments have recently emerged in the field of RNA therapeutics, with potential applications in the treatment of human diseases. The second International Conference on RNA in drug development was held to highlight several novel RNA-based technologies, including different approaches to silence gene expression, the broad range of diagnostic and therapeutic applications for aptamers, and the targeting of RNA with small molecules. Highlights of the meeting included the utilisation of RNA interference to silence genes, with applications for the treatment of both cancer and viral infections, and for systemic silencing of gene expression. Novel approaches to safer drug design using aptamers were presented, which would enable control of their therapeutic activity to be achieved with antidote oligonucleotides. Updates were also presented on the clinical and preclinical development of ribozymes and aptamers, including good progress in increasing the half-life of these molecules in serum.
Read
Martin L
ML
University of Oxford, Department of Clinical Pharmacology, Radcliffe Infirmary, Woodstock Road, Oxford, OX2 6HE, UK. martin.read@clinpharm.ox.ac.uk
Farrow
Paul J
PJ
Seymour
Leonard W
LW
D000818:Animals; D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use; D000998:Antiviral Agents / Q000494:pharmacology / Q000627:therapeutic use; D052157:Aptamers, Nucleotide / Q000627:therapeutic use; D015374:Biosensing Techniques; D017322:Clinical Trials, Phase II as Topic; D015195:Drug Design; D004353:Drug Evaluation, Preclinical; D005822:Genetic Vectors; D015658:HIV Infections / Q000188:drug therapy; D019698:Hepatitis C, Chronic / Q000188:drug therapy; D006801:Humans; D051379:Mice; D009369:Neoplasms / Q000188:drug therapy; D012313:RNA / Q000008:administration & dosage / Q000138:chemical synthesis / Q000737:chemistry / Q000494:pharmacology / Q000627:therapeutic use*; D034622:RNA Interference; D034741:RNA, Small Interfering; D014409:Tumor Necrosis Factor-alpha / Q000037:antagonists & inhibitors
D000970:Antineoplastic Agents; D000998:Antiviral Agents; D052157:Aptamers, Nucleotide; D034741:RNA, Small Interfering; D014409:Tumor Necrosis Factor-alpha; D012313:RNA