The identification and validation of disease targets requires in-depth understanding of processes related to life, both in health and disease. Ultimately, it requires the translation of an effect at the molecular level (genes, proteins, metabolites or drugs) to cellular networks. It also needs a multidisciplinary collaboration and integration of scientific disciplines. For example:
- Studying individual proteins experimentally, computationally and through bioinformatics approaches
- Cell biology and the role of metabolic, regulatory and signaling pathways in cell functioning
- Integrative and comparative approaches to understand the ultimate effects of intervention by drugs and therapeutics
Successful approaches to identify and validate disease targets therefore require a systems biology approach, integrating a broad range of disciplines into a cohesive network that can be probed deeply to understand the mechanisms of disease and drug effects.
This bottom-up approach to understanding biological processes culminates in target identification and validation. It is ideally complemented by an experimentally-driven top-down approach in which new factors involved in disease are identified through functional genomics, transcriptomics, proteomics, metabolomics, chemical genomics and advanced integrative statistics and bioinformatics.
Integrating bottom-up and top-down approaches – which are well represented in AIMMS – will yield innovative and much more effective strategies for target identification and validation.