A target is an enzyme or a receptor that may either block or promote a chemical reaction, thus interfering with the genesis or progression of a disease. To identify a potential target, scientists need to study the physiology and the biochemistry of the disease by breaking down all biochemical steps and identifying ligands, receptors, and all interactions leading to a specific condition. They also need to identify exactly where in the sequence it is possible to block or to promote a specific action.
To validate the target (4) scientists have to accumulate credible evidence. They must be able to induce an observable change to establish the relationship between the identified mechanism and its potential effect on the disease. The effect should be dose-dependant and specific, with a clearly defined mechanism of action. These effects are then tested in animal models, where any dangerous side effects may be identified.
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