Research ArticleGENE THERAPY
Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model
- Monica Nizzardo1,*,
- Chiara Simone1,
- Federica Rizzo1,
- Sabrina Salani1,
- Sara Dametti1,
- Paola Rinchetti1,
- Roberto Del Bo1,
- Kevin Foust2,
- Brian K. Kaspar2,3,4,
- Nereo Bresolin1,
- Giacomo P. Comi1 and
- Stefania Corti1
- 1Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
- 2Department of Neuroscience, The Ohio State University, Columbus, OH 43210, USA.
- 3The Research Institute at Nationwide Children’s Hospital, Columbus, OH 43205, USA.
- 4Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, OH 43210, USA.
- ↵*Corresponding author: E-mail: monica.nizzardo1{at}gmail.com
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Science Advances 13 Mar 2015:
Vol. 1, no. 2, e1500078
DOI: 10.1126/sciadv.1500078
Vol. 1, no. 2, e1500078
DOI: 10.1126/sciadv.1500078
Monica Nizzardo
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Chiara Simone
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Federica Rizzo
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Sabrina Salani
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Sara Dametti
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Paola Rinchetti
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Roberto Del Bo
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Kevin Foust
Department of Neuroscience, The Ohio State University, Columbus, OH 43210, USA.
Brian K. Kaspar
Department of Neuroscience, The Ohio State University, Columbus, OH 43210, USA.
The Research Institute at Nationwide Children’s Hospital, Columbus, OH 43205, USA.
Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, OH 43210, USA.
Nereo Bresolin
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Giacomo P. Comi
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Stefania Corti
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, and Neurology Unit, IRCCS Foundation Ca’ Granda, Ospedale Maggiore Policlinico, 20122 Milan, Italy.
