MR Dictionary

MR for drug targets

MR has increasingly been used within the field of drug target validation, testing for on- and off-target effects of a particular drug, whilst providing some evaluation of efficacy. Such an application requires knowledge about the drug target and biological pathway by which the drug influences a particular exposure.

The first examples of such applications occurred within the cardiovascular disease field, whereby inhibitors of certain pathways involved in low-density lipoprotein cholesterol synthesis - including inhibitors of cholesteryl ester transfer protein (CETP) and proprotein convertase subtilisin–kexin type 9 (PCSK9). By targeting the genes that code for those proteins or metabolites in an MR context, it is possible to mimic the activity of such drugs and estimate or predict the efficacy and off-target effects of such drugs before, during or after taken to trials in humans. Therefore, many drug companies are increasingly including such evaluations of drug targets using MR analyses (amongst other human genetic evidence) in the prioritisation of drugs.


Other terms in 'Definition of MR and study designs':