Prevention of Transthyretin Amyloid Disease by Changing Protein Misfolding Energetics

@article{Hammarstrm2003PreventionOT,
  title={Prevention of Transthyretin Amyloid Disease by Changing Protein Misfolding Energetics},
  author={Per Hammarstr{\"o}m and R. Luke Wiseman and Evan T. Powers and Jeffery W. Kelly},
  journal={Science},
  year={2003},
  volume={299},
  pages={713 - 716}
}
Genetic evidence suggests that inhibition of amyloid fibril formation by small molecules should be effective against amyloid diseases. Known amyloid inhibitors appear to function by shifting the aggregation equilibrium away from the amyloid state. Here, we describe a series of transthyretin amyloidosis inhibitors that functioned by increasing the kinetic barrier associated with misfolding, preventing amyloidogenesis by stabilizing the native state. The trans-suppressor mutation, threonine 119… 
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