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Since
the pathology of Huntington’s
disease (HD) is not well understood,
suitable molecular drug targets for
disease-modifying therapy have not been
identified yet. However, it is assumed
and widely accepted that proteolysis
of mutant huntingtin and aggregation
formation are crucial steps in disease
progression.
Thus, a ‘pathway screen’
(forward chemical genetics approach)
specific for mutant huntingtin is employed
for the identification of small molecule
inhibitors of huntingtin-mediated toxicity
in cellular systems. |
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