Research ArticleNIEMANN-PICK TYPE A DISEASE

Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

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Science Translational Medicine  21 Aug 2019:
Vol. 11, Issue 506, eaat3738
DOI: 10.1126/scitranslmed.aat3738

Article Information

vol. 11 no. 506

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History: 
  • Received for publication February 20, 2018
  • Resubmitted January 23, 2019
  • Accepted for publication July 29, 2019
  • .

Author Information

  1. Lluis Samaranch1,*,,
  2. Azucena Pérez-Cañamás2,*,
  3. Beatriz Soto-Huelin2,*,
  4. Vivek Sudhakar1,
  5. Jerónimo Jurado-Arjona2,
  6. Piotr Hadaczek1,
  7. Jesús Ávila2,
  8. John R. Bringas1,
  9. Josefina Casas3,
  10. Haifeng Chen4,
  11. Xingxuan He5,
  12. Edward H. Schuchman5,
  13. Seng H. Cheng6,
  14. John Forsayeth1,
  15. Krystof S. Bankiewicz1,, and
  16. María Dolores Ledesma2,
  1. 1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
  2. 2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
  3. 3RUBAM, IQAC-CSIC & CIBEREHD, 08034 Barcelona, Spain.
  4. 4Virovek Inc., Hayward, CA 94545, USA.
  5. 5Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
  6. 6Rare Disease, Sanofi, Framingham, MA 01701, USA.
  1. Corresponding author. Email: dledesma{at}cbm.csic.es (M.D.L.); krzysztof.bankiewicz{at}osumc.edu (K.S.B.)
  • * These authors contributed equally to this work.

  • Present address: Department of Neurological Surgery, The Ohio State University, Columbus, OH 43210, USA.

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