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Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice.
Mylvara AV, Gibson AL, Gu T, Davidson CD, Incao AA, Melnyk K, Pierre-Jacques D, Cologna SM, Venditti CP, Porter FD, Pavan WJ. Mylvara AV, et al. Among authors: pierre jacques d. bioRxiv [Preprint]. 2024 Jun 8:2024.06.07.597901. doi: 10.1101/2024.06.07.597901. bioRxiv. 2024. PMID: 38895471 Free PMC article. Preprint.