From Mechanism to Drug Target: Genetic Support for Therapeutic Hypotheses

Enrichment; the payoff that motivates the whole exercise.

Questions this Concept answers

Why does genetic support for a target's direction of effect increase confidence in a drug hypothesis compared to expression association alone?

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Using Genetic Evidence to Validate Drug Targets in Drug Discovery

In drug discovery, a "drug target" is a molecular entity (typically a gene/protein) chosen for therapeutic intervention, and "target validation" refers to the (informally defined) evidence supporting…