Developing MYOrganoids as a Translational Model to Evaluate Gene Therapy Products for DMD

Time: 9:30 am
day: Day One


  • Highlighting the urgent need to develop in vitro models mimicking human DMD phenotypes
  • Using 3D cultures and biomaterials to allow the reconstitution of tissue architecture and microenvironment essential for pathophysiological studies
  • Using DMD-derived MYOrganoids to evaluate the therapeutic efficiency of micro-dystrophin in proof-of-concept studies