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• W2890953289 endingPage "2025" @default.
• W2890953289 startingPage "2018" @default.
• W2890953289 abstract "We report the first evidence of GEX1A, a polyketide known to modulate alternative pre-mRNA splicing, as a potential treatment for Niemann-Pick type C disease. GEX1A was isolated from its producing organism, Streptomyces chromofuscus, and screened in NPC1 mutant cells alongside several semisynthetic analogues. We found that GEX1A and analogues are capable of restoring cholesterol trafficking in NPC1 mutant fibroblasts, as well as altering the expression of NPC1 isoforms detected by Western blot. These results, along with the compound’s favorable pharmacokinetic properties, highlight the potential of spliceosome-targeting scaffolds such as GEX1A for the treatment of genetic diseases." @default.
• W2890953289 created "2018-09-27" @default.
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• W2890953289 date "2018-09-06" @default.
• W2890953289 modified "2023-09-28" @default.
• W2890953289 title "GEX1A, a Polyketide from <i>Streptomyces chromofuscus</i>, Corrects the Cellular Defects Associated with Niemann-Pick Type C1 in Human Fibroblasts" @default.
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• W2890953289 doi "https://doi.org/10.1021/acs.jnatprod.8b00314" @default.
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