Pantethine depletes cystinotic fibroblasts of cystine
| Autor: | Martin Zatz, Jean DeB. Butler |
|---|---|
| Rok vydání: | 1983 |
| Předmět: |
medicine.medical_specialty
Pantetheine Cystinosis Cystine Cystamine chemistry.chemical_compound Internal medicine medicine Humans Sulfhydryl Compounds Cells Cultured Dose-Response Relationship Drug business.industry Pantethine Fanconi syndrome Fibroblasts medicine.disease Endocrinology chemistry Pantetheinase Pediatrics Perinatology and Child Health Cysteamine business |
| Zdroj: | The Journal of pediatrics. 102(5) |
| ISSN: | 0022-3476 |
| Popis: | CYSTINOSIS is an autosomal recessive genetic disease in which cystine characteristically accumulates in the lysosomes of cells. The organ most affected is the kidney; the Fanconi syndrome develops early in life, and is followed by progressive renal failure, t The disease is presumably the consequence of the abnormally high cystine concentrations, 2 so therapy has been directed toward reducing the cystine content of the patient's ceils. The most effective agent for accomplishing this goal has been cysteamine, 3 and this compound is currently being used for the treatment of cystinosis. In cultured fibroblasts, cystamine, the disulfide form of cysteamine, will lower intracellular cystine as effectively as cysteamine. 4 The therapeutic value of cysteamine is reduced by its disagreeable taste and odor and low therapeutic index? However, most analogues that lack these drawbacks are ineffective. Phosphocysteamine, which is about half as toxic as cysteamine and lacks the objectionable odor, has been proposed as a substitute for cysteamine. 6 Cysteamine is an endogenous substance, and we hypothesized that it might be possible to promote its formation by cells in situ, as needed. The only known source of intracellular cysteamine is the action of the enzyme pantetheinase on pantetheine, 7 a reaction involved in coenzyme A metabolism. Cystinotic cells have normal levels of pantethei |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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