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Article

Phenanthridin-6-one derivatives as the first class of non-steroidal pharmacological chaperones for Niemann-Pick disease type C1 protein.

Bioorganic & medicinal chemistry letters - 15 Jun 2017

Fukuda Hiromitsu, Karaki Fumika, Dodo Kosuke, Noguchi-Yachide Tomomi, Ishikawa Minoru, Hashimoto Yuichi, Ohgane Kenji

Abstract excerpt

Niemann-Pick disease type C is a fatal, progressive neurodegenerative disease mostly caused by mutations in Nieamnn-Pick type C1 (NPC1), a late endosomal membrane protein that is essential for intracellular cholesterol transport. The most prevalent mutation, I1061T (Ile to Thr), interferes with the protein folding process. Consequently, mutated but intrinsically functional NPC1 proteins are prematurely degraded...

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Source

Identifiers

PMID: 28465104
DOI: 10.1016/j.bmcl.2017.04.062

Genes

NPC1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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