Type C Niemann-Pick disease fibroblasts and their transformed cell lines are hypersensitive to HMG-CoA reductase inhibitors.
The deficiency of exogenous cholesterol transport within fibroblasts of Niemann-Pick disease type C (NPC) has been addressed. In this report we confirmed that the endogenous synthesis of cholesterol within cells was markedly increased in NPC fibroblasts and those transformed by origin-defective simian virus 40. The transformed fibroblasts and those of the primary culture were hypersensitive to 3-hydroxy-3-methylglutaryl-coenzyme A reductase inhibitors as a consequence of their dependence on endogenous cholesterol biosynthesis. The transformed fibroblasts should help further biochemical and genetic research in this condition.
Yamamoto
T
T
Department of Pediatrics, Jikei University, School of Medicine, Tokyo, Japan.
Ohashi
T
T
Tokoro
T
T
Maekawa
K
K
Eto
Y
Y
D002461:Cell Line, Transformed; D005347:Fibroblasts / Q000187:drug effects* / Q000201:enzymology; D006801:Humans; D006903:Hydroxymethylglutaryl CoA Reductases / Q000378:metabolism; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D009542:Niemann-Pick Diseases / Q000201:enzymology*
D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D006903:Hydroxymethylglutaryl CoA Reductases