Duchenne Muscular Dystrophy Myogenic Cells from Urine-Derived Stem Cells Recapitulate the Dystrophin Genotype and Phenotype

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 specimen...

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Veröffentlicht in:	Human gene therapy 2016-10, Vol.27 (10), p.772-783
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Dystrophin - genetics Exons - genetics Genetic Therapy Genotype Humans Muscle Development - genetics Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - pathology Muscular Dystrophy, Duchenne - therapy Oligonucleotides, Antisense - genetics Oligonucleotides, Antisense - therapeutic use Phenotype Stem Cells - metabolism
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