herboxidiene and Niemann-Pick-Disease--Type-C

herboxidiene has been researched along with Niemann-Pick-Disease--Type-C* in 1 studies

Other Studies

1 other study(ies) available for herboxidiene and Niemann-Pick-Disease--Type-C

ArticleYear
GEX1A, a Polyketide from Streptomyces chromofuscus, Corrects the Cellular Defects Associated with Niemann-Pick Type C1 in Human Fibroblasts.
    Journal of natural products, 2018, 09-28, Volume: 81, Issue: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. 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.

    Topics: Cell Line; Cholesterol; Fatty Alcohols; Fibroblasts; Humans; Molecular Structure; Niemann-Pick Disease, Type C; Polyketides; Protein Biosynthesis; Pyrans; Streptomyces

2018