Research ArticleAmyotrophic Lateral Sclerosis

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

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Science Translational Medicine  31 Oct 2018:
Vol. 10, Issue 465, eaau6414
DOI: 10.1126/scitranslmed.aau6414

Article Information

vol. 10 no. 465

PubMed: 
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History: 
  • Received for publication January 28, 2018
  • Resubmitted July 1, 2018
  • Accepted for publication October 11, 2018
  • .

Author Information

  1. Florie Borel1,2,*,
  2. Gwladys Gernoux1,*,
  3. Huaming Sun1,
  4. Rachel Stock1,
  5. Meghan Blackwood1,
  6. Robert H. Brown Jr.3, and
  7. Christian Mueller1,4,
  1. 1Horae Gene Therapy Center, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.
  2. 2Shire, 125 Binney Street, Cambridge, MA 02142, USA.
  3. 3Department of Neurology, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.
  4. 4Department of Pediatrics, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.
  1. Corresponding author. Email: chris.mueller{at}umassmed.edu (C.M.); robert.brown{at}umassmed.edu (R.H.B.)
    • * These authors contributed equally to this work as co-first authors.

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