• Dipender Gill, Marie-Joe Dib, Héléne T Cronjé, Ville Karhunen, Benjamin Woolf, Eloi Gagnon, Iyas Daghlas, Michael Nyberg, Donald Drakeman, and Stephen Burgess.
• Sequoia Genetics, London, UK. dipender.gill@sequoiagenetics.com.
• Bmc Med. 2024 Oct 15; 22 (1): 473473.

BackgroundDrug target Mendelian randomization describes the use of genetic variants as instrumental variables for studying the effects of pharmacological agents. The paradigm can be used to inform on all aspects of drug development and has become increasingly popular over the last decade, particularly given the time- and cost-efficiency with which it can be performed even before commencing clinical studies.Main BodyIn this review, we describe the recent emergence of drug target Mendelian randomization, its common pitfalls, how best to address them, as well as potential future directions. Throughout, we offer advice based on our experiences on how to approach these types of studies, which we hope will be useful for both practitioners and those translating the findings from such work.ConclusionsDrug target Mendelian randomization is nuanced and requires a combination of biological, statistical, genetic, epidemiological, clinical, and pharmaceutical expertise to be utilized to its full potential. Unfortunately, these skillsets are relatively infrequently combined in any given study.© 2024. The Author(s).

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