ERC Starting Grant · 2017
Skeletal muscle is the most abundant human tissue and contains mainly post-mitotic nuclei. It also expresses the largest gene known in nature – dystrophin – whose mutations cause Duchenne muscular dystrophy, the most frequent and incurable childhood muscle disorder. These characteristics create hurdles that negatively impact on the development of therapies for muscle diseases, ranging from acute tissue loss to chronic neuromuscular disorders. Moreover, a lack of humanised models of muscle regeneration delays the understanding of its regenerative dynamics. My work has pioneered the use of human induced pluripotent stem (iPS) cells to generate genetically corrected myogenic cells for the…
From the public funding record at EU CORDIS. Describes the funded project, not the reviews below.