Article
Duchenne Muscular Dystrophy Myogenic Cells from Urine-Derived Stem Cells Recapitulate the Dystrophin Genotype and Phenotype.
Human gene therapy - 1 Oct 2016
Falzarano Maria Sofia, D'Amario Domenico, Siracusano Andrea, Massetti Massimo, Amodeo Antonio, La Neve Federica, Maroni Camilla Reina, Mercuri Eugenio, Osman Hana, Scotton Chiara, Armaroli Annarita, Rossi Rachele, Selvatici Rita, Crea Filippo, Ferlini Alessandra
Abstract excerpt
A ready source of autologous myogenic cells is of vital importance for drug screening and functional genetic studies in Duchenne muscular dystrophy (DMD), a rare disease caused by a variety of dystrophin gene mutations. As stem cells (SCs) can be easily and noninvasively obtained from urine specimens, we set out to determine whether they could be myogenically induced and useful in DMD research. To this end, we...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.