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Screening of small molecule chemical library identifies leads that reduce cholesterol accumulation in human NPC1 fibroblasts

2022-11-16

Abstract excerpt

Although considerable progress has been made in finding a therapy for Niemann-Pick type C1 (NPC1) disease, there still is no effective approved treatment. Previously, we reported results from a small molecule screen that was conducted on Chinese Hamster Ovary (CHO) cells (CT60) that lacked NPC1 expression. The hit compounds that were effective in reducing cholesterol accumulation in CT60 cells were not effective o...

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Identifiers and source

Literature Corpus work
4633cc94-28e6-500b-8cca-bdac74c4f4b8
DOI
10.1101/2022.11.16.516777
Open publication

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Screening of small molecule chemical library identifies leads that reduce cholesterol accumulation in human NPC1 fibroblastsDOI 10.1101/2022.11.16.516777
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