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Updated: Nov 14, 2025

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Candidate Gene Testing in Clinical Cohort Studies with Multiplexed Genotyping and Mass Spectrometry
Published on: June 21, 2018
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Genomics-driven drug discovery based on disease-susceptibility genes
Kyuto Sonehara1, Yukinori Okada2,3,4
1Department of Statistical Genetics, Osaka University Graduate School of Medicine, 2-2 Yamadaoka, Suita, 565-0871, Japan.
Inflammation and Regeneration
|March 11, 2021
Summary
Genetics-led drug discovery uses gene insights to find new medicines and predict side effects. Advances focus on drug repurposing, Mendelian randomization, and omics data for better clinical translation.
Area of Science:
- Genetics
- Pharmacology
- Bioinformatics
Background:
- Genome-wide association studies (GWAS) have identified numerous disease-susceptibility genes.
- Translating genetic discoveries into clinical practice remains a significant challenge.
- Identifying effective drug targets and predicting side effects are crucial for successful clinical trials.
Purpose of the Study:
- To review recent advances in genetics-led drug discovery.
- To explore future perspectives in this field.
- To highlight key methodologies like drug repurposing, Mendelian randomization, and omics data integration.
Main Methods:
- Review of recent scientific literature.
- Focus on genetics-led drug discovery approaches.
- Discussion of drug repurposing strategies.
- Explanation of Mendelian randomization principles.
- Integration of multifaceted omics data.
Main Results:
- Accumulating knowledge of gene-disease associations.
- Identification of challenges in translating genetic findings to clinical practice.
- Highlighting the potential of genetics to guide drug target identification.
- Demonstrating the utility of drug repurposing, Mendelian randomization, and omics data.
Conclusions:
- Genetics-led drug discovery offers a promising avenue for developing effective therapeutics.
- Overcoming challenges in clinical translation requires innovative approaches.
- The integration of diverse data types, including omics data, is essential for advancing the field.
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