My Work
The work that we do in my lab is multidisciplinary. I received my PhD in physical chemistry and I did my post-doctorate work in synthesis. In the lab we study ribonucleic acid (RNA) and how to use it as a target for drugs. Unlike deoxyribonucleic acid (DNA), which forms a double helix, RNA is single stranded and it folds onto itself often times in strange and unusual ways. Every single protein in a body is encoded by RNA.
In my lab we try to capitalize on my experience in both physical chemistry, in particular RNA structure and thermodynamics, and in chemical synthesis to understand what types of ligands like to bind RNA and better use it as a target for drugs. In general, we test 10,000 compounds for binding to 10,000 or so different RNAs. For each compound, we find what RNA structures it likes to bind. These preferences are searched for in genomes to target RNAs that cause disease.
Designing drugs that target RNA is still a relatively new process. But the potential applications are huge. RNA can cause cancer, genetic diseases, and bacterial and viral infections. We hope to develop methods to rationally design drugs targeting RNAs that cause each of these diseases. We are especially interested in targeting RNAs from orphan diseases to initiate the pharmaceutical industry into "adopting" them.
My Research
RNA is an important biomolecule that encodes and catalyzes the synthesis of protein. RNAs (microRNAs) also play important regulatory roles in cells that, if altered, can contribute to cancer and other diseases. These factors make RNA an important target for the design of therapeutics. At present, however, few therapeutics target RNA. This is due to a limited understanding of how to target RNA with a small molecule.
The research in the Disney lab focuses on developing a better understanding of how to target RNA with drug-like compounds. Our ultimate goal is to develop a general set of rules, or code, that can be used to design a therapeutic towards an RNA using only its sequence.
Our research focuses on three main areas:
- Development of new high throughput screening assays to understand the binding of small molecules to RNA;
- Development of two-dimensional combinatorial screening to probe RNA and chemical space simultaneously to identify features in both governing molecular recognition; and
- Use information from these studies to target RNAs that cause disease. Currently, we are targeting pathogenic RNAs to which there are no known treatments.