Date: April 11, 2023
Time: 8:00am (PDT), 11:00am (EDT), 5:00pm (CEST)
Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. Muscle fibers lacking dystrophin are more sensitive to damage and over time muscle tissue and muscle function is progressively lost resulting in wheelchair dependency around age 10 and premature death in the 2nd to 4th decade of life. Mutations that maintain the reading frame allow the production of internally deleted, partially functional dystrophins. These mutations are associated with the later onset, less progressive Becker muscular dystrophy. This reading frame rule forms the premise of current dystrophin restoring therapies, such as exon skipping to allow Duchenne patients to produce Becker-type dystrophin and gene therapy aiming to deliver a micro-dystrophin transgene to muscles.
In this lecture I will provide context to the different approaches, outline considerations to the question of how much dystrophin is enough, how to measure this in the clinical trial settings, and how we employed the ProteinSimple capillary immunoassay for analyzing dystrophin in our preclinical studies.
- Summarize the criteria for clinically relevant dystrophin restoration
- Assess the methods for quantifying dystrophin restoration in clinical and pre-clinical settings
- Explain how Simple Western is used for reproducible dystrophin quantification
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