RNA, A Powerful Molecule

The entire human genetic code is contained in our DNA in the nucleus of every cell. DNA, packaged into genes on chromosomes, has all the information for protein production. Proteins are responsible for cell development and maintenance. RNAs have many functions within the cell. Some carry a copy of the instructions for protein production from DNA to the machinery that produces proteins. Other RNAs are part of that machinery.

Scientists are now finding that cells make countless other RNAs to control protein production by acting as the on-off switch for genes. Human disease results when proteins are not made correctly or in the quantity required by the cell. There are many steps in protein production. An error at any step can lead to disease. However, a century of pharmaceutical development has not targeted the steps to protein production, only the end product of disease, the incorrectly made protein. RNA, as a novel therapeutic drug class, can address problems at any step of protein production. This approach has an unlimited ability to treat the most difficult of human diseases.

The power to treat and cure disease using RNA’s ability to harness and control genes enables simultaneous identification of disease causes, treatments and possible cures. The research programs at the Institute develop and apply novel tools to validate RNA as targets for drug design. Institute researchers extend these programs into drug discovery by designing, synthesizing and testing the delivery, toxicity and efficacy of candidate therapeutics to destroy or neutralize the disease-causing RNA function.