Research ArticleNIEMANN-PICK TYPE A DISEASE
Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A
- View ORCID ProfileLluis Samaranch1,*,†,
- View ORCID ProfileAzucena Pérez-Cañamás2,*,
- View ORCID ProfileBeatriz Soto-Huelin2,*,
- View ORCID ProfileVivek Sudhakar1,
- Jerónimo Jurado-Arjona2,
- Piotr Hadaczek1,
- View ORCID ProfileJesús Ávila2,
- John R. Bringas1,
- View ORCID ProfileJosefina Casas3,
- Haifeng Chen4,
- View ORCID ProfileXingxuan He5,
- View ORCID ProfileEdward H. Schuchman5,
- Seng H. Cheng6,
- John Forsayeth1,
- Krystof S. Bankiewicz1,†,‡ and
- View ORCID ProfileMaría Dolores Ledesma2,‡
- 1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
- 2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
- 3RUBAM, IQAC-CSIC & CIBEREHD, 08034 Barcelona, Spain.
- 4Virovek Inc., Hayward, CA 94545, USA.
- 5Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
- 6Rare Disease, Sanofi, Framingham, MA 01701, USA.
- ↵‡Corresponding author. Email: dledesma{at}cbm.csic.es (M.D.L.); krzysztof.bankiewicz{at}osumc.edu (K.S.B.)
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Science Translational Medicine 21 Aug 2019:
Vol. 11, Issue 506, eaat3738
DOI: 10.1126/scitranslmed.aat3738
Vol. 11, Issue 506, eaat3738
DOI: 10.1126/scitranslmed.aat3738
Lluis Samaranch
1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
Azucena Pérez-Cañamás
2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
Beatriz Soto-Huelin
2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
Vivek Sudhakar
1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
Jerónimo Jurado-Arjona
2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
Piotr Hadaczek
1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
Jesús Ávila
2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
John R. Bringas
1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
Josefina Casas
3RUBAM, IQAC-CSIC & CIBEREHD, 08034 Barcelona, Spain.
Haifeng Chen
4Virovek Inc., Hayward, CA 94545, USA.
Xingxuan He
5Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Edward H. Schuchman
5Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Seng H. Cheng
6Rare Disease, Sanofi, Framingham, MA 01701, USA.
John Forsayeth
1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
Krystof S. Bankiewicz
1Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA.
María Dolores Ledesma
2Centro Biologia Molecular Severo Ochoa (CSIC-UAM), 28049 Madrid, Spain.
