As an ISCRM REU intern, Catlyn Wetzel will employ CRISPR/Cas9 gene editing technologies to develop a promoterless gene trap cell line. Dr. Bengtsson’s lab is working on gene therapies for Duchenne muscular dystrophy (DMD), a genetic disorder which leads to muscle breakdown and eventual heart failure, caused by a mutation in the dystrophin gene. Dystrophin, a protein necessary for muscle functioning and structure, is vital in shock absorption and stabilization of the muscle. In DMD, the gene encoding for dystrophin is non-functional, meaning dystrophin is not produced; muscles quickly break down and cannot repair fast enough. This cell line can be used to monitor and track expression of the DMD gene without requiring a loss-of-function mutation, and it can be used throughout the lab to test the potential effectiveness of certain new therapies.
