Screening of small molecule chemical library identifies leads that reduce cholesterol accumulation in human NPC1 fibroblasts
Nina H. Pipalia, Amy Y. Huang, Harold Ralph, Frederick R. Maxfield
The paper identifies small molecule compounds that reduce cholesterol accumulation in human NPC1 mutant fibroblasts but not in NPC1-/- cells, suggesting on-target effects.
bioRxiv · Nov 16, 2022 · PreprintNeuroscience