The present invention is directed to a novel, target-blind approach to
drug discovery. The concept is to model human phenotypes in a teleost,
such as a zebrafish, and then screen compounds, e.g., small molecules,
for their ability to alter the phenotype. Because the screen is performed
with a whole vertebrate organism and uses a phenotype as the output, the
need to first identify target genes is eliminated. This approach is
powerful because a single screen can theoretically detect drugs affecting
any target relevant to the phenotype being observed, even if those
targets are not yet characterized.
