ReviewCold Spring Harbor perspectives in medicine2022
Human Genomics and Drug Development.
Review in Cold Spring Harbor perspectives in medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
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
25 citing papers in PubMed, 1 synthesis or guideline pooled it, 39 citations in OpenAlex.
- Genetic evidence for serum amyloid P component as a drug target in neurodegenerative disorders.Open biology · 2024Pooled it
- Mendelian randomisation in ophthalmology: An overview and practical guide for clinicians.European journal of ophthalmology · 2026Review
- The immunoproteome and multimorbidity: A Mendelian randomization study.Science advances · 2026Article
- Drug targets for lipid modification and risk of type 2 diabetes: a cis-Mendelian randomization study.Cardiovascular diabetology · 2026Article
- Assessment of the value of polygenic risk scores in the prevention of disease.Journal of medical screening · 2025Review
- Phosphodiesterase-5 Inhibition and Alzheimer's Disease Risk: A Mendelian Randomisation Study.Aging cell · 2025Article
- Potential Drug Targets for Migraine Identified Through Mendelian Randomization Analysis of the Human Plasma Proteome.Molecular neurobiology · 2025Article
- Druggable Targets for Postpartum Depression: A Mendelian Randomization and Colocalization Study.Cellular and molecular neurobiology · 2025Article
- Proteome-Wide Mendelian Randomization Identifies Candidate Causal Proteins for Cardiovascular Diseases.Advanced genetics (Hoboken, N.J.) · 2025Article
- A multi-omics target study for glioblastoma multiforme (GBM) based on Mendelian randomization analysis.IBRO neuroscience reports · 2025Article
- A new paradigm for drug discovery in the treatment of complex diseases: drug discovery and optimization.Chinese medicine · 2025Review
- Genetic association of lipid-lowering drug target genes with pancreatic cancer: a Mendelian randomization study.Scientific reports · 2025Article
- Strategies for the drug development of cancer therapeutics.Frontiers in pharmacology · 2025Review
- Mendelian Randomisation: Concepts, Opportunities, Challenges, and Future Directions.Global heart · 2025Review
- Integrating metabolomics and proteomics to identify novel drug targets for heart failure and atrial fibrillation.Genome medicine · 2024Article
- Repurposing anti-osteoporosis drugs for autoimmune diseases: A two-sample Mendelian randomization study.Heliyon · 2024Article
- Review
- Comparing the effects of CETP in East Asian and European ancestries: a Mendelian randomization study.Nature communications · 2024Article
- Identifying novel proteins for migraine by integrating proteomes from blood and CSF with genome-wide association data.CNS neuroscience & therapeutics · 2024Article
- The Role of Genetics in Advancing Cardiometabolic Drug Development.Current atherosclerosis reports · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
Abstract
Insights into the genetic basis of human disease are helping to address some of the key challenges in new drug development including the very high rates of failure. Here we review the recent history of an emerging, genomics-assisted approach to pharmaceutical research and development, and its relationship to Mendelian randomization (MR), a well-established analytical approach to causal inference. We demonstrate how human genomic data linked to pharmaceutically relevant phenotypes can be used for (1) drug target identification (mapping relevant drug targets to diseases), (2) drug target validation (inferring the likely effects of drug target perturbation), (3) evaluation of the effectiveness and specificity of compound-target engagement (inferring the extent to which the effects of a compound are exclusive to the target and distinguishing between on-target and off-target compound effects), and (4) the selection of end points in clinical trials (the diseases or conditions to be evaluated as trial outcomes). We show how genomics can help identify indication expansion opportunities for licensed drugs and repurposing of compounds developed to clinical phase that proved safe but ineffective for the original intended indication. We outline statistical and biological considerations in using MR for drug target validation (drug target MR) and discuss the obstacles and challenges for scaled applications of these genomics-based approaches.
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.