Research ArticleGene Therapy

Long-Term Follow-Up After Gene Therapy for Canavan Disease

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Science Translational Medicine  19 Dec 2012:
Vol. 4, Issue 165, pp. 165ra163
DOI: 10.1126/scitranslmed.3003454

Article Information

vol. 4 no. 165 165ra163

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication November 14, 2011
  • Accepted for publication November 30, 2012

Author Information

  1. Paola Leone1,*,
  2. David Shera2,
  3. Scott W.J. McPhee3,
  4. Jeremy S. Francis1,
  5. Edwin H. Kolodny4,
  6. Larissa T. Bilaniuk5,
  7. Dah-Jyuu Wang5,
  8. Mitra Assadi6,
  9. Olga Goldfarb6,
  10. H. Warren Goldman6,
  11. Andrew Freese7,
  12. Deborah Young8,
  13. Matthew J. During8,9,
  14. R. Jude Samulski3,10 and
  15. Christopher G. Janson7,11
  1. 1Department of Cell Biology, Cell & Gene Therapy Center, University of Medicine & Dentistry of New Jersey, Stratford, NJ 08034, USA.
  2. 2Division of Biostatistics, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  3. 3Asklepios Biopharmaceuticals, Chapel Hill, NC 27517, USA.
  4. 4Department of Neurology, New York University School of Medicine, New York, NY 10016, USA.
  5. 5Division of Neuroradiology, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  6. 6Cooper Neurological Institute, Camden, NJ 08103, USA.
  7. 7Department of Neurosurgery, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
  8. 8Departments of Molecular Medicine and Pathology, and Clinical Pharmacology, University of Auckland, Auckland 1023, New Zealand.
  9. 9Departments of Neuroscience and Molecular Virology, Immunology & Medical Genetics, Ohio State University, Columbus, OH 43210, USA.
  10. 10Department of Pharmacology, Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599, USA.
  11. 11Department of Neurology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
  1. *To whom correspondence should be addressed. E-mail: leonepa{at}umdnj.edu

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