GEX1A, a Polyketide from Streptomyces chromofuscus, Corrects the Cellular Defects Associated with Niemann-Pick Type C1 in Human Fibroblasts.
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ABSTRACT:
SUBMITTER: Granatosky EA
PROVIDER: S-EPMC6868502 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
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GEX1A, a Polyketide from Streptomyces chromofuscus, Corrects the Cellular Defects Associated with Niemann-Pick Type C1 in Human Fibroblasts.
Granatosky Eve A EA DiPrimio Nina N Pickering Jarred R E JRE Stevens D Cole DC Perlstein Ethan O EO Taylor Richard E RE
Journal of natural products 20180906 9
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. ...[more]
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