Target Validation
The process of establishing that modulating a specific molecular target will produce a therapeutic benefit in the disease of interest. Employs multiple orthogonal approaches including genetic knockdown/knockout, chemical tool compounds, human genetic evidence (e.g., GWAS, Mendelian genetics), and animal models. Strong target validation significantly increases the probability of clinical success.
أمثلة
- PCSK9 loss-of-function mutations validating LDL-lowering target
- CRISPR knockout confirming oncogene dependency
- Human genetics linking BACE1 to Alzheimer's pathology
هل تعلم؟
The process of establishing that modulating a specific molecular target will produce a therapeutic benefit in the disease of interest. Employs multiple orthogonal approaches including genetic knockdown/knockout, chemical tool compounds, …