Tetrahydrobiopterin shows chaperone activity for tyrosine hydroxylase

J Neurochem. 2008 Jul;106(2):672-81. doi: 10.1111/j.1471-4159.2008.05423.x. Epub 2008 Apr 14.

Abstract

Tyrosine hydroxylase (TH) is the rate-limiting enzyme in the synthesis of catecholamine neurotransmitters. Primary inherited defects in TH have been associated with l-DOPA responsive and non-responsive dystonia and infantile parkinsonism. In this study, we show that both the cofactor (6R)-l-erythro-5,6,7,8-tetrahydrobiopterin (BH(4)) and the feedback inhibitor and catecholamine product dopamine increase the kinetic stability of human TH isoform 1 in vitro. Activity measurements and synthesis of the enzyme by in vitro transcription-translation revealed a complex regulation by the cofactor including both enzyme inactivation and conformational stabilization. Oral BH(4) supplementation to mice increased TH activity and protein levels in brain extracts, while the Th-mRNA level was not affected. All together our results indicate that the molecular mechanisms for the stabilization are a primary folding-aid effect of BH(4) and a secondary effect by increased synthesis and binding of catecholamine ligands. Our results also establish that orally administered BH(4) crosses the blood-brain barrier and therapeutic regimes based on BH(4) supplementation should thus consider the effect on TH. Furthermore, BH(4) supplementation arises as a putative therapeutic agent in the treatment of brain disorders associated with TH misfolding, such as for the human TH isoform 1 mutation L205P.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Biopterins / administration & dosage
  • Biopterins / analogs & derivatives*
  • Brain / drug effects
  • Brain / metabolism*
  • Circular Dichroism / methods
  • Dopamine / administration & dosage
  • Dopamine / metabolism*
  • Female
  • Humans
  • Isoenzymes / metabolism
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Protein Binding
  • Protein Biosynthesis / drug effects
  • Protein Biosynthesis / physiology
  • Protein Folding
  • Tyrosine 3-Monooxygenase / genetics
  • Tyrosine 3-Monooxygenase / metabolism*

Substances

  • Isoenzymes
  • Biopterins
  • Tyrosine 3-Monooxygenase
  • sapropterin
  • Dopamine