Emerging Developments in RNA Therapeutics

Abstract

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.

A versatile reducible polycation-based system for efficient delivery of a broad range of nucleic acids

“…One reason for this has been the recent emergence of small interfering RNA (siRNA) to trigger RNA interference with many potential therapeutic applications (46). In addition, there is considerable interest in delivering mRNA to dendritic cells to stimulate potent CTL responses and induce antitumour immunity (47).…”

RNA-based therapeutic strategies for cancer

“…From day 8 -29 a daily subcutaneous bolus injection was given. The results from this trial are expected in 2004 [18].…”

“…A significant twofold reduction in tumour volume has so far been shown following SIRPLEX 120-N administered intraperitoneally at a dose of 200 µg/kg/day for 29 days in nude mice subcutaneously implanted with human pancreatic adenocarcinomas. Effective tumour reduction of malignant melanoma was also observed in nude mice following administration of SIRPLEX 120-N in combination with the chemotherapeutic agent dacarbazine (3 x 80 mg/kg/day) [18].…”