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Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.
Rodriguez-Gil JL, Baxter LL, Watkins-Chow DE, Johnson NL, Davidson CD, Carlson SR, Incao AA; NISC Comparative Sequencing Program; Wallom KL, Farhat NY, Platt FM, Dale RK, Porter FD, Pavan WJ. Rodriguez-Gil JL, et al. Among authors: incao aa. Hum Mol Genet. 2021 Nov 30;30(24):2456-2468. doi: 10.1093/hmg/ddab194. Hum Mol Genet. 2021. PMID: 34296265 Free PMC article.
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: incao aa. 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.
Genetic background modifies phenotypic severity and longevity in a mouse model of Niemann-Pick disease type C1.
Rodriguez-Gil JL, Watkins-Chow DE, Baxter LL, Elliot G, Harper UL, Wincovitch SM, Wedel JC, Incao AA, Huebecker M, Boehm FJ, Garver WS, Porter FD, Broman KW, Platt FM, Pavan WJ. Rodriguez-Gil JL, et al. Among authors: incao aa. Dis Model Mech. 2020 Mar 13;13(3):dmm042614. doi: 10.1242/dmm.042614. Dis Model Mech. 2020. PMID: 31996359 Free PMC article.