Decreased intracellular ATP content and intact mitochondrial energy generating capacity in human cystinotic fibroblasts

Pediatr Res. 2006 Feb;59(2):287-92. doi: 10.1203/01.pdr.0000196334.46940.54.

Abstract

Cystinosis is an autosomal recessive lysosomal storage disorder caused by a defect in the lysosomal cystine carrier cystinosin. Cystinosis is the most common cause of inherited Fanconi syndrome leading to renal failure, in which the pathogenesis is still enigmatic. Based on studies of proximal tubules loaded with cystine dimethyl ester (CDME), altered mitochondrial adenosine triphosphate (ATP) production was proposed to be an underlying pathologic mechanism. Thus far, however, experimental evidence supporting this hypothesis in humans is lacking. In this study, energy metabolism was extensively investigated in primary fibroblasts derived from eight healthy subjects and eight patients with cystinosis. Patient's fibroblasts accumulated marked amounts of cystine and displayed a significant decrease in intracellular ATP content. Remarkably, overall energy-generating capacity, activity of respiratory chain complexes, ouabain-dependent rubidium uptake reflecting Na,K-ATPase activity, and bradykinin-stimulated mitochondrial ATP production were all normal in these cells. In conclusion, the data presented demonstrate that mitochondrial energy-generating capacity and Na,K-ATPase activity are intact in cultured cystinotic fibroblasts, thus questioning the idea of altered mitochondrial ATP synthesis as a keystone for the pathogenesis of cystinosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenosine Triphosphate / biosynthesis
  • Adenosine Triphosphate / metabolism*
  • Case-Control Studies
  • Cystinosis / enzymology
  • Cystinosis / metabolism*
  • Cystinosis / pathology
  • Electron Transport
  • Fibroblasts / enzymology
  • Fibroblasts / metabolism
  • Humans
  • Mitochondria / metabolism*
  • Sodium-Potassium-Exchanging ATPase / metabolism

Substances

  • Adenosine Triphosphate
  • Sodium-Potassium-Exchanging ATPase