Protein therapy for Unverricht-Lundborg disease using cystatin B transduction by TAT-PTD. Is it that simple?

Epilepsy Res. 2006 Nov;72(1):75-9. doi: 10.1016/j.eplepsyres.2006.07.009. Epub 2006 Aug 22.

Abstract

In this work we analysed the characteristics of the cell-permeable peptide TAT-PTD fused to cystatin B (CSTB) to evaluate its potential for protein therapy of Unverricht-Lundborg (UL) epilepsy. TAT-PTD-CSTB does not penetrate the cells despite initial evidence of time and concentration-dependent transduction. Therefore, it cannot be used as a form of replacement of the intracytoplasmic protein missing in UL. Importantly, we discuss precautions to avoid false-positive results when working with TAT-PTD for protein therapy of neurological diseases.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Blood-Brain Barrier
  • Cell Membrane / physiology
  • Cystatin B
  • Cystatins / genetics
  • Cystatins / metabolism*
  • Cystatins / therapeutic use
  • Cysteine Proteinase Inhibitors
  • Gene Products, tat / genetics
  • Gene Products, tat / metabolism*
  • Gene Products, tat / therapeutic use
  • Humans
  • Plasmids
  • Protein Binding
  • Protein Transport
  • Transduction, Genetic*
  • Unverricht-Lundborg Syndrome / therapy*

Substances

  • CSTB protein, human
  • Cystatins
  • Cysteine Proteinase Inhibitors
  • Gene Products, tat
  • Cystatin B