Pharmacological chaperone therapy for lysosomal storage disorders - leveraging aspects of the folding pathway to maximize activity of misfolded mutant proteins

FEBS J. 2007 Oct;274(19):4943. doi: 10.1111/j.1742-4658.2007.06043.x.
No abstract available

Publication types

  • Introductory Journal Article

MeSH terms

  • Humans
  • Lysosomal Storage Diseases / drug therapy*
  • Lysosomal Storage Diseases / metabolism
  • Mutation
  • Protein Folding