ReviewCambridge prisms. Precision medicine2023
Mendelian randomization as a tool to inform drug development using human genetics.
Review in Cambridge prisms. Precision medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
37 citing papers in PubMed, 59 citations in OpenAlex.
- Genetic Insights Into Protective Effects of GLP-1 Receptor Agonists on Stroke and Alzheimer Disease Across Ancestries.Neurology. Genetics · 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
- Identifying druggable proteins of the association of chronotype on breast cancer using Mendelian randomization.Communications medicine · 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
- Genetic variation in antidiabetic drug targets: associations with Parkinson's disease risk and age at onset.NPJ Parkinson's disease · 2026Article
- A Mendelian randomization-based drug repurposing pipeline: application to lipid traits and coronary artery disease.medRxiv : the preprint server for health sciences · 2026Article
- From early-onset asthma to chronic obstructive pulmonary disease: potential mediating proteins and therapeutic targets.Briefings in bioinformatics · 2026Article
- Moving Mendelian Randomization From Traditional Risk Factors to Molecular Targets for Drug Development and Clinical Trials in Nephrology.Kidney international reports · 2026Review
- Erythropoietin drug targets and cancer risk: A two-sample Mendelian randomization study.Medicine · 2026Article
- Integrative mendelian randomization approaches for therapeutic target prioritisation in immune-mediated diseases.Scientific reports · 2026Article
- Genetic Evidence Links Gestational Diabetes Mellitus to Increased ER-Negative Breast Cancer Risk Through a Mendelian Randomization Analysis.International journal of endocrinology · 2026Article
- Genetic pharmacoepidemiology of JAK inhibitors in chronic immune-mediated skin diseases: implications for precision therapy and medication safety.Frontiers in pharmacology · 2026Article
- Multi-omics integration identifies PGAP3 as a tumor-intrinsic factor associated with CD8Frontiers in molecular biosciences · 2026Article
- Integrated expression quantitative trait loci and Mendelian randomization analyses of the candidate genes and pathways identified for myocardial infarction.Frontiers in molecular biosciences · 2026Article
- Integrative multi-omics Mendelian randomization and functional validation identifies RNASET2 as a novel therapeutic target for autoimmune thyroiditis.Frontiers in endocrinology · 2026Article
- Genetic Downregulation of Interleukin-6 Signaling, Coagulation Function, and Risk of Thromboembolic Disease.Journal of the American Heart Association · 2025Article
- Therapeutic Targets for Sepsis: Multicenter Proteome-Wide Analyses and Experimental Validation.Journal of proteome research · 2025Article
- Application of Human Genetics to Prioritize Coagulation Cascade Protein Targets for Ischemic Stroke Prevention.Stroke · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Drug development is essential to the advancement of human health, however, the process is slow, costly, and at high risk of failure at all stages. A promising strategy for expediting and improving the probability of success in the drug development process is the use of naturally randomized human genetic variation for drug target identification and validation. These data can be harnessed using the Mendelian randomization (MR) analytic paradigm to proxy the lifelong consequences of genetic perturbations of drug targets. In this review, we discuss the myriad applications of the MR paradigm for human drug target identification and validation. We review the methodology and applications of MR, key limitations of MR, and potential future opportunities for research. Throughout the review, we refer to illustrative examples of MR analyses investigating the consequences of genetic inhibition of interleukin 6 signaling which, in some cases, have anticipated results from randomized controlled trials. As human genetic data become more widely available, we predict that MR will serve as a key pillar of support for drug development efforts.
Indexed as
Identifiers
What OpenQuestion holds
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.