Proteins are a group of macromolecular molecules that are necessary for the body to function properly. We could not exist without them. In light of their obvious importance, my lab revolves around understanding them on a molecular basis. My research has encompassed various aspects of protein biology, ranging from isolating proteins from slugs and yeast to deciphering their roles in human diseases. I take an interdisciplinary approach to investigating scientific questions, incorporating biology, as well as chemical and biochemical approaches. Hence, my research spans across various scientific fields.
My research over the past several years has focused on deciphering how various proteins play a role in blood coagulation and cancer progression. Specifically, my lab has been actively developing RNA-based therapeutic molecules for treating individuals with blood-related diseases. The RNA molecules we developed are termed “RNA aptamers.” Aptamers are single-stranded nucleic acids, either DNA or RNA, that bind to their target protein with high affinity and specificity. The high affinity binding of aptamers is accomplished by their folding into a three dimensional structure specific to the target protein. In general, aptamers alter protein function by binding to and inducing a conformational change in the protein, which subsequently disrupts function. The direct inhibition of protein function by aptamers suggests that they are similar to monoclonal antibodies. In 2004, the US Food and Drug Administration approved the first RNA aptamer for use against age-related macular degeneration. There are several advantages to using RNA aptamers as therapeutic agents, including: 1) they exhibit high affinity and specific binding to their target protein, 2) they are mostly non-immunogenic, 3) they are synthesized with 2’fluropyrimidines to ensure plasma stability, and 4) their half-life in plasma can be increased when they are conjugated to high molecular weight polyethylene glycol or cholesterol esters. Despite the success of aptamers in biomedical research, they also play a role in bio-imaging, parasitic biology, and as anti-venom agents. Currently, we are working on several projects, including developing RNA aptamer to treat individuals with sickle cell disease.