We are now on the verge of viewing effector molecules and other
regulatory sites as therapeutic targets for the amelioration of
human and animal disease. The recognition, for example, that mutant
proteins are frequently misrouted molecules, rather than
functionally defective ones, changes our approach to "inborn errors
of metabolism" and offers new approaches for pharmacological
discovery, based on rescue of receptors, ion channels and enzymes
with pharmacoperones. Ion channels, regulators of G-protein
signaling and enzymes engaged in regulation, now present
opportunities for drug development.
The state of our art also benefits by the availability of
superior tools that allow measurement of interactions and afford
unprecedented insight into the biomolecular interactions that
present novel approaches to drug design.
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