Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells (pages 6045–6060)
Mirella Meregalli, Claire Navarro, Clementina Sitzia, Andrea Farini, Erica Montani, Nicolas Wein, Paola Razini, Cyriaque Beley, Letizia Cassinelli, Daniele Parolini, Marzia Belicchi, Dario Parazzoli, Luis Garcia and Yvan Torrente
Article first published online: 8 OCT 2013 | DOI: 10.1111/febs.12523
Miyoshi myopathy (MM) is a fatal muscle disease caused by mutations in the dysferlin gene. We developed, for the first time, a strategy based on the use of lentiviral vector which express the full-length dysferlin in blood-derived CD133+ stem cells isolated from MM patients. Transplantation of these cells into animal models of dysferlinopathy allowed the rescue of dystrophic muscle fibers.