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Webinar: Rapid and Reproducible Quantification of Full-Length Dystrophin and Micro-Dystrophin

Webinar Summary

Duchenne muscular dystrophy (DMD), the most lethal musculoskeletal disease, is caused by mutations in the X-linked gene dystrophin. Patients show delayed motor milestone development at 2-6 years of age and are wheelchair-bound by their early teenage years.  Novel gene replacement therapy using micro-dystrophin has shown great promise to reduce the disease burden of DMD in preclinical studies and human trials.  An important aspect in validating micro-dystrophin therapy is to quantify and compare the amount of micro-dystrophin expressed in dystrophic muscles after treatment to that of full-length dystrophin in normal subjects. We implemented the Simple Western™ technique to evaluate micro-dystrophin expression in 80 different muscle samples collected following systemic micro-dystrophin gene transfer, as well as full-length dystrophin from normal muscles. We conclude that Simple Western™ is an excellent technical platform for rapid and reproducible quantification of full-length dystrophin and micro-dystrophin.



Dennis Pérez-López

Doctoral Candidate,
University of Missouri-Columbia