PM.20.006 – Building Muscle Strength
Cardiac and skeletal muscle diseases are frequently caused by a genetic defect, which is present at birth and affects entire families. Thereby inherited muscle diseases pose an enormous economic and societal burden as affected individuals suffer from disability at young age, leading to loss of healthy years, inability to work, and reliance on family and health care. Identification of carriers of a genetic defect enables timely, preventive treatment strategies based on personalized medicine. However, currently, no effective preventive and curative therapies are available for progressive muscle diseases. The lack of therapies is due to the fact that current research models do not adequately mimic human disease. This hampers drug testing.
Building Muscle Strength (BMS) creates a knowledge and expertise chain in which patient data and biobanks are linked with stem cell-derived human muscle models. These models mimic muscle disease in patients, and enable to test efficacy of personalized therapies. Subsequently, trial-readiness drugs can be tested in the well-characterized patient groups. In addition to the development and implementation of game-changing therapies for severe muscle disorders, and a coincident reduction of societal and economic burden, we anticipate that BMS will reduce the number of animals used for research on muscle disease.
Big Data, high-throughput screening, individual variability, Inherited muscle diseases, iPSC-derived models
|Organisation||Amsterdam UMC, location Vrije Universiteit (VUMC)|
|Name||Prof. dr. J. (Jolanda) van der Velden|