Utilizing In Vitro 3D Muscle Bundles to Assess the Functionality of AAV Therapeutics for Duchenne Muscular Dystrophy (DMD)
Time: 2:15 pm
day: Day One
Details:
- Reproducible assay to test gene therapy candidates in DMD-patient derived cells
- Repair of truncated dystrophin at the sarcolemma of DMD-treated myotubes
- Structural restoration of the DAPC complex in DMD myotubes treated with an AAV-mediated exon skipping
