Ontology highlight
ABSTRACT:
ORGANISM(S): Homo sapiens
SUBMITTER: Stefania Corti
PROVIDER: E-GEOD-27207 | biostudies-arrayexpress |
REPOSITORIES: biostudies-arrayexpress
Corti Stefania S Nizzardo Monica M Simone Chiara C Falcone Marianna M Nardini Martina M Ronchi Dario D Donadoni Chiara C Salani Sabrina S Riboldi Giulietta G Magri Francesca F Menozzi Giorgia G Bonaglia Clara C Rizzo Federica F Bresolin Nereo N Comi Giacomo P GP
Science translational medicine 20121201 165
Spinal muscular atrophy (SMA) is among the most common genetic neurological diseases that cause infant mortality. Induced pluripotent stem cells (iPSCs) generated from skin fibroblasts from SMA patients and genetically corrected have been proposed to be useful for autologous cell therapy. We generated iPSCs from SMA patients (SMA-iPSCs) using nonviral, nonintegrating episomal vectors and used a targeted gene correction approach based on single-stranded oligonucleotides to convert the survival mo ...[more]