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BACKGROUND Amyotrophic lateral sclerosis (ALS) is a progressive, lethal, neurodegenerative disease, currently without any effective therapy. Multiple advantages make mesenchymal stromal cells (MSC) a… (More)
BACKGROUND Mesenchymal stromal cells (MSC) are attractive candidates for the treatment of Duchenne muscular dystrophy (DMD) but how the donor MSC distribute in multiple organs and whether the… (More)
SCN1A is the most relevant epilepsy gene. Mutations of SCN1A generate phenotypes ranging from the extremely severe form of Dravet syndrome (DS) to a mild form of generalized epilepsy with febrile… (More)