Research ArticleGENE THERAPY

Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model

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Science Advances  13 Mar 2015:
Vol. 1, no. 2, e1500078
DOI: 10.1126/sciadv.1500078

Article Information

vol. 1 no. 2

Online ISSN: 
History: 
  • Received for publication January 20, 2015
  • Accepted for publication February 15, 2015
  • .

Author Information

  1. Monica Nizzardo1,*,
  2. Chiara Simone1,
  3. Federica Rizzo1,
  4. Sabrina Salani1,
  5. Sara Dametti1,
  6. Paola Rinchetti1,
  7. Roberto Del Bo1,
  8. Kevin Foust2,
  9. Brian K. Kaspar2,3,4,
  10. Nereo Bresolin1,
  11. Giacomo P. Comi1 and
  12. Stefania Corti1
  1. 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.
  2. 2Department of Neuroscience, The Ohio State University, Columbus, OH 43210, USA.
  3. 3The Research Institute at Nationwide Children’s Hospital, Columbus, OH 43205, USA.
  4. 4Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, OH 43210, USA.
  1. *Corresponding author: E-mail: monica.nizzardo1{at}gmail.com

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