Summary
This narrative review synthesises the principles of Mendelian randomisation design and its application in cardiovascular epidemiology. The authors explain how genetic variation can serve as a natural experiment to strengthen causal inference from observational data, analogous to treatment randomisation in RCTs, and demonstrate growing applications in drug efficacy prediction and repurposing. The review emphasises that valid MR results depend critically on justified assumptions about the genetic variants employed as instrumental variables.
UK applicability
As a methodological review of a statistical design approach, the findings are globally applicable to UK cardiovascular research and clinical trial design. UK-based researchers and the NHS may apply MR approaches to inform drug development and repurposing strategies for cardiovascular risk factors relevant to the UK population.
Key measures
Mendelian randomisation methodology; genetic variants as instrumental variables; causal inference assumptions; cardiovascular risk factors and disease outcomes
Outcomes reported
The study describes principles and applications of Mendelian randomisation (MR) design for establishing causal relationships between cardiometabolic risk factors and cardiovascular diseases. It reviews how MR has been applied to predict drug efficacy and safety and to explore drug repurposing potential.
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