Researchers from the Institute of Myology and Généthon report pioneering work in the treatment of Duchenne muscular dystrophy (DMD):
- they started from the observation that the muscle fibres of DMD patients and their murine counterparts showed lysosomal abnormalities,
- which are also accompanied by overexpression of Galectin-3, a key protein in lysosomal function.
By using trehalose, a lysosome-protective disaccharide, in addition to gene therapy (micro-dystrophin), they achieved much greater efficacy of the latter.
