Development of a muscle -on-chip model for Pompe disease and facioscapulohumeral muscular dystrophy

Project summary

Animal models or human cells in tissue culture often do not respond to medication as cells in intact organs in the human body would do. This consortium is developing miniature muscles that will aid in the testing and development of existing and novel therapies for Pompe disease and facioscapulohumeral muscular dystrophy.

The deliverables of this project will be the generation of a set of cell models based on stem cells that contain mutations causing Pompe disease or facioscapulohumeral muscular dystrophy; the generation of small muscle bundles from these models and their incorporation in a chip that allows measurements of their strength; incorporation of technology in the chip that allows the monitoring of additional parameters for disease progression; and measurements of these disease parameters to monitor human disease symptoms in the muscle-on-chip platform.