Structural and mechanistic basis of the interaction between a pharmacological chaperone and human phenylalanine hydroxylase

Chembiochem. 2012 Jun 18;13(9):1266-9. doi: 10.1002/cbic.201200188. Epub 2012 Apr 30.


Not without a chaperone: Pharmacological chaperones are designed to bind and ideally stabilise their target protein. Here, we elucidate the molecular mechanism of a potential pharmacological chaperone to treat phenylketonuria. The crystal structure of human phenylalanine hydroxylase with compound IV may help in the rational design of more efficient compounds to treat this disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Kinetics
  • Models, Molecular
  • Phenylalanine Hydroxylase / chemistry*
  • Phenylalanine Hydroxylase / metabolism*
  • Protein Binding
  • Protein Multimerization / drug effects
  • Protein Refolding / drug effects
  • Protein Structure, Secondary
  • Small Molecule Libraries / metabolism*
  • Small Molecule Libraries / pharmacology*


  • Small Molecule Libraries
  • Phenylalanine Hydroxylase