ReviewWellcome open research2021
Mendelian randomization for studying the effects of perturbing drug targets.
Review in Wellcome open research, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 163 papers, 8 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
163 citing papers in PubMed, 8 syntheses or guidelines pooled it.
- Impact of Glucagon-like Peptide-1 Receptor Agonists on Mental Illness: Evidence from a Mendelian Randomization Study.International journal of molecular sciences · 2025Pooled it
- The genomic architecture of circulating cytokine levels points to drug targets for immune-related diseases.Communications biology · 2025Pooled it
- Pooled it
- HMG-CoA reductase is a potential therapeutic target for migraine: a mendelian randomization study.Scientific reports · 2024Pooled it
- Pooled it
- Mendelian Randomization Analysis of Genetic Proxies of Thiazide Diuretics and the Reduction of Kidney Stone Risk.JAMA network open · 2023Pooled it
- Repurposing antidiabetic drugs for rheumatoid arthritis: results from a two-sample Mendelian randomization study.European journal of epidemiology · 2023Pooled it
- Therapeutic potential of IL6R blockade for the treatment of sepsis and sepsis-related death: A Mendelian randomisation study.PLoS medicine · 2023Pooled it
- Proteome-wide genetic analyses identify ASGR1 as a potential biomarker linking smoking with ischemic heart disease.Nature cardiovascular research · 2026Article
- Sex-aware causal inference assessment of the immune system in complex neurodegenerative diseases.Brain : a journal of neurology · 2026Article
- Long-Term Efficacy and Safety of GLP-1R Agonist and SGLT2 Inhibitor Therapy in the General Population: A Mendelian Randomization Study.Circulation. Genomic and precision medicine · 2026Article
- Investigating the repurposing potential of immune checkpoint inhibition for cancer treatment using Mendelian randomisation.Research square · 2026Article
- From GWAS Signals to Molecular Mechanisms: Explainable AI for Causal Gene Prioritization and Biomolecular Target Interpretation.Biomolecules · 2026Review
- Comparison of methods for assessing effects of risk factors on disease progression in Mendelian randomization under index event bias.American journal of human genetics · 2026Article
- An integrative mendelian randomisation and drug mechanism framework for target prioritisation and therapeutic repurposing in major depression.Translational psychiatry · 2026Article
- Leveraging Mendelian randomization to inform drug discovery and development for ischemic stroke.Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism · 2026Review
- Genomic Evidence on Antihypertensive Drug Targets and Oral Disease Outcomes.Journal of periodontal research · 2026Article
- The role of lipid-lowering drugs in urologic and male reproductive cancers: a Mendelian randomization study.Translational andrology and urology · 2026Article
- Genetic variation in antidiabetic drug targets: associations with Parkinson's disease risk and age at onset.NPJ Parkinson's disease · 2026Article
- Article
103 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
18 authors.
Funding
Abstract
Drugs whose targets have genetic evidence to support efficacy and safety are more likely to be approved after clinical development. In this paper, we provide an overview of how natural sequence variation in the genes that encode drug targets can be used in Mendelian randomization analyses to offer insight into mechanism-based efficacy and adverse effects. Large databases of summary level genetic association data are increasingly available and can be leveraged to identify and validate variants that serve as proxies for drug target perturbation. As with all empirical research, Mendelian randomization has limitations including genetic confounding, its consideration of lifelong effects, and issues related to heterogeneity across different tissues and populations. When appropriately applied, Mendelian randomization provides a useful empirical framework for using population level data to improve the success rates of the drug development pipeline.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.