Michael Mohsen, PhD
Developing a New Type of Gene Therapy With Broad Application Potential
Summary
Gene therapy aims to treat or prevent disease by fixing the underlying genetic problem. My research aims to develop a new type of gene therapy that could be implemented to treat a broad range of diseases, including cancers and genetic disorders. First, I will evaluate small molecule drugs and drug-like compounds as candidates for this strategy by considering their safety in humans and bioavailability, or the extent to which they enter circulation and reach their target destination in the body. Next, I will generate novel receptors made of RNA (molecules essential in regulating genes) that selectively recognize the target compounds. These receptors will then be employed in the construction of “designer riboswitches,” RNA sensors that determine which genes to turn “on” and “off” in human cells via administration of a drug-like compound. Designer riboswitches represent a new modality in gene therapy that could be applied widely and with great therapeutic outlook.
The PhRMA Foundation is the first organization to financially support my research in my scientific career. In science, experiments often don’t work, grants aren’t funded, and manuscripts are rejected. This only makes the successes more memorable. Thanks to the PhRMA Foundation for believing in my research!