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Transcriptome of 2-Hydroxypropyl-Beta-Cyclodextrin Treatment in Niemann-Pick Disease Type C1

Transcriptome of HPβCD-treated Niemann-pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.

Jorge L. Rodriguez-Gil, L. Baxter, D. Watkins-Chow, et al. (2021). Human molecular genetics. Cited 19 times. https://doi.org/10.1093/hmg/ddab194