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

Duchenne muscular dystrophy (DMD), the most lethal musculoskeletal disease, is caused by mutations in the X-linked gene dystrophin. Patients with DMD 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.

In this GEN webinar, we will hear how Simple Western™ was used 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. Moreover, we will hear how Simple Western is an excellent analytical tool for rapid and reproducible quantification of micro-dystrophin and full-length dystrophin, the largest known human gene.

A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelist.

Produced with support from: