High throughput mini muscles
In this project miniature muscles will be developed on a chip from patient-derived stem cells that can be used for high throughput screening of drugs for muscle disorders. The consortium consists of two academic partners, Erasmus University MC and Leiden University MC, and the company Optics11. The academic partners contribute stem cell models, while the company provides the technology for measuring contractile forces of the muscles.
There are over 600 human disorders known that affect skeletal muscle, but for only very few a therapy is available. This has a large impact on human society. An important reason is the lack of suitable model systems that faithfuly represent human muscle disease and that can be used to develop and test potential treatment options.
Patient-derived stem cells will be used that mimic the patient in a dish. These cells will be used to generate small muscles that can contract and are grown on a chip that can read out the contractile force. By generating multiple mini muscles that can be measured simultaneously, we generate a platform that allows large-scale testing. This will be instrumental for the development of novel therapies for human muscle disorders.
The deliverables include the generation of stem cell models that contain fluorescent genes that report muscle maturation; the generation of a platform on which 96 mini muscles can be grown and for which contractile force can be measured at the same time; and testing of the platform and application to two muscle disorders: Pompe disease and facioscapulohumeral muscular dystrophy. The endresult is a platform for high throughput screening of therapies for human muscle disorders using human, contractile mini muscles grown in 3D.