Using genetic association data to guide drug discovery and development: Review of methods and applications.

ABSTRACT: Evidence on the validity of drug targets from randomized trials is reliable but typically expensive and slow to obtain. In contrast, evidence from conventional observational epidemiological studies is less reliable because of the potential for bias from confounding and reverse causation. Mendelian randomization is a quasi-experimental approach analogous to a randomized trial that exploits naturally occurring randomization in the transmission of genetic variants. In Mendelian randomization, genetic variants that can be regarded as proxies for an intervention on the proposed drug target are leveraged as instrumental variables to investigate potential effects on biomarkers and disease outcomes in large-scale observational datasets. This approach can be implemented rapidly for a range of drug

SUBMITTER: Burgess S 

PROVIDER: S-EPMC9943784 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature