Evaluating the efficacy and safety of existing targeted drugs using genomic data
This study aims to discover the causal effects of a drug that either enhances or represses the function of a gene or protein. To do so, we will leverage a statistical method called Mendelian randomization. In this method, the genomic variants located in the gene will be used to mimic the known effect of the drug. The participants in the UK Biobank will be grouped according to how many effect alleles they have in these genomic variants. For example, the participants with 1 or more effect alleles will be thought as the medication group (who took the drug), while the participants without an effect allele will be though as the control group or placebo group. Since the UK Biobank is a deep phenotyping resource, we would have the opportunity of investigating many clearly defined diseases simultaneously. By observing the differences in the proportion of these disease outcomes between the groups, we can tell if the drug is protective or risk for them.