ArticleNature communications2021
Identifying therapeutic drug targets using bidirectional effect genes.
Article in Nature communications, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
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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
18 citing papers in PubMed, 30 citations in OpenAlex.
- Functional analysis of NPR2 variants supports the therapeutic rationale for CNP in short stature.American journal of human genetics · 2026Article
- A scalable framework for identifying allelic series from summary statistics.American journal of human genetics · 2025Article
- Smooth muscle expression of RNA editing enzyme ADAR1 controls activation of the RNA sensor MDA5 in atherosclerosis.Nature cardiovascular research · 2025Article
- The genetic basis of human height.Nature reviews. Genetics · 2025Review
- Combining genetic proxies of drug targets and time-to-event analyses from longitudinal observational data to identify target patient populations.BMC cardiovascular disorders · 2025 · on this mapObservational
- Evaluation of enzyme activity predictions for variants of unknown significance in Arylsulfatase A.Human genetics · 2025Article
- Identification and functional analysis of NPR2 truncating mutations in two Chinese families with short stature.BMC pediatrics · 2025Article
- Evaluation of enzyme activity predictions for variants of unknown significance in Arylsulfatase A.bioRxiv : the preprint server for biology · 2024Article
- Effects of SPI1-mediated transcriptome remodeling on Alzheimer's disease-related phenotypes in mouse models of Aβ amyloidosis.Nature communications · 2024Article
- Better understanding the phenotypic effects of drugs through shared targets in genetic disease networks.Frontiers in pharmacology · 2024Article
- Development of a human genetics-guided priority score for 19,365 genes and 399 drug indications.Nature genetics · 2024Article
- Genetic and Gene Expression Resources for Osteoporosis and Bone Biology Research.Current osteoporosis reports · 2023Review
- Pharmacological and Genetic Disruption of C-Type Natriuretic Peptide (International journal of molecular sciences · 2023Article
- Identification of novel genes includingFrontiers in endocrinology · 2023Article
- Target Discovery for Drug Development Using Mendelian Randomization.Methods in molecular biology (Clifton, N.J.) · 2022Review
- Common genetic variants with fetal effects on birth weight are enriched for proximity to genes implicated in rare developmental disorders.Human molecular genetics · 2021Article
- Identification and Tissue-Specific Characterization of Novel SHOX-Regulated Genes in Zebrafish Highlights SOX Family Members Among Other Genes.Frontiers in genetics · 2021Article
- Identification of Immune-Related Genes Associated With Bladder Cancer Based on Immunological Characteristics and Their Correlation With the Prognosis.Frontiers in genetics · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Prioritizing genes for translation to therapeutics for common diseases has been challenging. Here, we propose an approach to identify drug targets with high probability of success by focusing on genes with both gain of function (GoF) and loss of function (LoF) mutations associated with opposing effects on phenotype (Bidirectional Effect Selected Targets, BEST). We find 98 BEST genes for a variety of indications. Drugs targeting those genes are 3.8-fold more likely to be approved than non-BEST genes. We focus on five genes (IGF1R, NPPC, NPR2, FGFR3, and SHOX) with evidence for bidirectional effects on stature. Rare protein-altering variants in those genes result in significantly increased risk for idiopathic short stature (ISS) (OR = 2.75, p = 3.99 × 10
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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.