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

Lluis Samaranch, Azucena Pérez-Cañamás, Beatriz Soto-Huelin, Vivek Sudhakar, Jerónimo Jurado-Arjona, Piotr Hadaczek, Jesús Ávila, John R. Bringas, Josefina Casas, Haifeng Chen, Xingxuan He, Edward H. Schuchman, Seng H. Cheng, John Forsayeth, Krystof S. Bankiewicz, María Dolores Ledesma

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Medicine & Life Sciences