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 o f Niemann-Pick disease type C (NPC) has been addressed. In this report we confirmed that the endogenous synthesis of cholesterol within cell s was markedly increased in NPC fibroblasts and those transformed by o rigin-defective simian virus 40. The transformed fibroblasts and those of the primary culture were hypersensitive to 3-hydroxy-3-methylgluta ryl-coenzyme A reductase inhibitors as a consequence of their dependen ce on endogenous cholesterol biosynthesis. The transformed fibroblasts should help further biochemical and genetic research in this conditio n.