Research ArticleGene Therapy

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

See allHide authors and affiliations

Science Translational Medicine  29 Nov 2017:
Vol. 9, Issue 418, eaam6375
DOI: 10.1126/scitranslmed.aam6375

Article Information

vol. 9 no. 418

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication December 23, 2016
  • Accepted for publication June 13, 2017

Author Information

  1. Francesco Puzzo1,2,*,
  2. Pasqualina Colella1,*,
  3. Maria G. Biferi3,
  4. Deeksha Bali4,
  5. Nicole K. Paulk5,
  6. Patrice Vidal1,3,
  7. Fanny Collaud1,
  8. Marcelo Simon-Sola1,3,
  9. Severine Charles1,
  10. Romain Hardet3,
  11. Christian Leborgne1,
  12. Amine Meliani1,3,
  13. Mathilde Cohen-Tannoudji3,
  14. Stephanie Astord3,
  15. Bernard Gjata1,
  16. Pauline Sellier1,3,
  17. Laetitia van Wittenberghe1,
  18. Alban Vignaud1,
  19. Florence Boisgerault1,
  20. Martine Barkats3,
  21. Pascal Laforet6,
  22. Mark A. Kay5,
  23. Dwight D. Koeberl7,
  24. Giuseppe Ronzitti1, and
  25. Federico Mingozzi1,3,
  1. 1INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.
  2. 2Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy.
  3. 3University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
  4. 4Biochemical Genetics Laboratory, Duke University Health System, Durham, NC 27710, USA.
  5. 5Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  6. 6Paris-Est Neuromuscular Center, Pitié-Salpêtrière Hospital and Raymond Poincaré Teaching Hospital, Garches, APHP, Paris, France.
  7. 7Division of Medical Genetics, Department of Pediatrics and Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC 27710, USA.
  1. Corresponding author. Email: gronzitti{at}genethon.fr (G.R.); fmingozzi{at}genethon.fr (F.M.)
    • * These authors contributed equally to this work.

    Altmetric

    Article usage

    Article usage: November 2017 to January 2018

    AbstractFullPdf
    Nov 2017262451117
    Dec 20174864130285
    Jan 20183074185