Lovastatin and sodium phenylacetate normalize the levels of very long chain fatty acids in skin fibroblasts of X‐ adrenoleukodystrophy

FEBS Letters

Khan, Mushfiquddin¹  Singh, Inderjit¹  Pahan, Kalipada¹ 

[1]Department of Pediatrics, Medical University of South Carolina, 171 Ashley Avenue, Charleston, SC 29425, USA

关键词: Lovastatin;    Phenylacetate;    Adrenoleukodystrophy;    Very long chain fatty acid;    Beta-oxidation;   

DOI  :  10.1016/S0014-5793(98)00370-6

学科分类：生物化学/生物物理

来源: John Wiley & Sons Ltd.

【 摘 要 】

The present study underlines the importance of lovastatin, an inhibitor of 3-hydroxy-3-methyl-glutaryl-coenzyme A (HMG-CoA) reductase, and the sodium salt of phenylacetic acid (NaPA), an inhibitor of mevalonate pyrophosphate decarboxylase, in normalizing the pathognomonic accumulation of saturated very long chain fatty acids (VLCFA) in cultured skin fibroblasts of X-adrenoleukodystrophy (X-ALD) in which the ALD gene is either mutated or deleted. Lovastatin or NaPA alone or in combination stimulated the β-oxidation of lignoceric acid (C_24:0) and normalized the elevated levels of VLCFA in skin fibroblasts of X-ALD. Ability of lovastatin and NaPA to normalize the pathognomonic accumulation of VLCFA in skin fibroblasts of X-ALD may identify these drugs as possible therapeutics for X-ALD.

【 授权许可】

Unknown