RNA Therapeutics

Our Research programs all seek to understand basic medical and biological functions, and disease mechanisms, with an eye towards the delineation of new and novel therapeutic targets. Many of us work on therapeutics related to those targets as well.

Our new focus on RNA Therapeutics has great potential. More and more scientists are discovering unknown roles for RNA in biology and in disease mechanisms. For instance, it is becoming accepted that microRNAs (mRNAs), small, non-coding RNAs that act on target genes at many levels, play important regulatory roles and that they are transferred in the body from cell to cell via vesicles called exosomes. Using exosomes and exosome-like vesicles to transfer mRNAs and inhibitory RNAs with therapeutic potential has great promise. Currently the top 100 drugs target about 40 proteins, many of these are receptors that are deemed “targetable.” However, there are around 30,000 protein-encoding genes in humans and perhaps another 4000-6000 non-coding genes that may have critical effects in regulating biology and therefore might be therapeutic targets. RNA therapeutics offer the possibility to modulate all of these targets and the potential to easily design, test and develop new agents. RNA therapeutics have already been approved by FDA (e.g. Mipomersen for hypercholesterolemia) and others are under development. We are at the beginning of a revolution in this field. In addition to non-coding RNAs, we now know that RNA is “edited,” which can mean that a protein’s amino acid sequence is different from that indicated by the encoding gene. This may provide new targets for diseases in which this process is altered and has implications for drugs that act at protein sites that are modified. The upshot is that new developments in our understanding of biology provide opportunities to develop new classes of therapeutics; we are pushing to take advantage of the advances in RNA biology and are among the pioneers in this area. 


W. Keith Jones, Ph.D. Exosomal RNA in Cardiovascular disease and regeneration.

Monsheel Sodhi, Ph.D. RNA editing in Neuropsychological disease.

Simon Kaja, Ph.D.  Exosomes and miRNA in optic nerve biology.