Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: A dose-response study in mice and nonhuman primates

Meyer, K., Ferraiuolo, L., Schmelzer, L. et al. (11 more authors) (2014) Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: A dose-response study in mice and nonhuman primates. Molecular Therapy, 23 (3). 477 -487. ISSN 1525-0016

Abstract

Metadata

Authors/Creators:
  • Meyer, K.
  • Ferraiuolo, L.
  • Schmelzer, L.
  • Braun, L.
  • McGovern, V.
  • Likhite, S.
  • Michels, O.
  • Govoni, A.
  • Fitzgerald, J.
  • Morales, P.
  • Foust, K.D.
  • Mendell, J.R.
  • Burghes, A.H.M.
  • Kaspar, B.K.
Copyright, Publisher and Additional Information: © The American Society of Gene & Cell Therapy
Dates:
  • Published: 9 December 2014
  • Accepted: 24 October 2014
Institution: The University of Sheffield
Academic Units: The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > Department of Neuroscience (Sheffield)
Depositing User: Symplectic Sheffield
Date Deposited: 13 Jan 2016 15:52
Last Modified: 09 Mar 2016 15:04
Published Version: http://dx.doi.org/10.1038/mt.2014.210
Status: Published
Publisher: Nature Publishing Group
Refereed: Yes
Identification Number: https://doi.org/10.1038/mt.2014.210

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