Researchers at Généthon and the Institute of Myology* have developed muscle organoids, called ‘MYOrganoids’, that reproduce Duchenne muscular dystrophy (DMD) in order to study gene therapy in greater detail.
- The ‘MYOrganoids’ were obtained from skeletal muscle cells derived from induced pluripotent stem cells from DMD patients co-cultured with fibroblasts from patients to accelerate their structural and contractile maturation while reproducing muscle weakness and fibrosis.
- Tested within these three-dimensional structures, microdystrophin gene therapy improved muscle function but only partially restored the glycoprotein complex components associated with dystrophin and did little to reduce fibrosis.
- An antifibrotic treatment could improve the effectiveness of gene therapy.
* Riyad El-Khoury and Guy Brochier – Morphological Unit at the Neuromuscular Investigation Center
Anne Bigot – Cellular and molecular orchestration in muscle regeneration, during ageing and in pathologies team at the Center of Research in Myology
