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Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice.
Davidson CD, Gibson AL, Gu T, Baxter LL, Deverman BE, Beadle K, Incao AA, Rodriguez-Gil JL, Fujiwara H, Jiang X, Chandler RJ, Ory DS, Gradinaru V, Venditti CP, Pavan WJ. Davidson CD, et al. Among authors: gu t. Life Sci Alliance. 2021 Aug 18;4(10):e202101040. doi: 10.26508/lsa.202101040. Print 2021 Oct. Life Sci Alliance. 2021. PMID: 34407999 Free PMC article.
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: gu t. bioRxiv [Preprint]. 2024 Jun 8:2024.06.07.597901. doi: 10.1101/2024.06.07.597901. bioRxiv. 2024. PMID: 38895471 Free PMC article. Preprint.
1,911 results