Alyson Fiorillo, Ph.D.
- Principal Investigator, Center for Genetic Medicine Research
Right before I started grad school, I volunteered at a Muscular Dystrophy Association Camp where I worked with hundreds of kids with muscular dystrophies and other neuromuscular disorders. This experience forever changed my life. It single handedly changed the course of my research career, one in which I am committed, passionate and unwavering towards the goal of finding viable therapeutics to improve the lives of kids with muscle diseases.
My laboratory has a strong focus on Duchenne Muscular Dystrophy (DMD), a progressive muscle wasting disease caused by the absence of dystrophin protein. To restore dystrophin, a personalized medicine approach called exon skipping has been given conditional approval for treatment of DMD patients. However, the amount of dystrophin restoration that occurs through exon skipping is inconsistent, and patchy. In our laboratory we employ a variety of techniques to understand and improve dystrophin rescue in DMD. This includes investigating microRNAs that target and downregulate dystrophin, understanding the crosstalk between muscle and the immune system, determining how a secondary dystrophin deficiency might impact other muscle disorders and characterizing novel models of microRNA deletion that my affect the pathophysiology of muscle disorders.