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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. Hum Mol Genet. 2021 Nov 30;30(24):2456-2468. doi: 10.1093/hmg/ddab194. Hum Mol Genet. 2021. PMID: 34296265 Free PMC article.