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

1. Samaranch, L.
2. Pérez-Cañamás, A.
3. Soto-Huelin, B.
4. Sudhakar, V.
5. Jurado-Arjona, J.
6. Hadaczek, P.
7. Ávila, J.
8. Bringas, J.R.
9. Casas, J.
10. Chen, H.
11. He, X.
12. Schuchman, E.H.
13. Cheng, S.H.
14. Forsayeth, J.
15. Bankiewicz, K.S.
16. Ledesma, M.D.