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About the Lab

The Fiorillo 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 may affect the pathophysiology of muscle disorders. 

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