A knock down strategy for rapid, generic, and versatile modelling of muscular dystrophies in 3D-tissue-engineered-skeletal muscle.
Stijn L M In 't GroenMarnix FrankenTheresa BockMarcus KrügerJessica C de GreefW W M Pim Pijnappel
Published in: Skeletal muscle (2024)
These results show that isogenic shRNA-based humanized 3D-TESM models provide a fast, cheap, and efficient tool to model muscular dystrophies and are useful for the preclinical evaluation of novel therapies.