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Using Human Genetics to Drive Drug Discovery: A Perspective
1Merck & Co, Inc, Boston, MA.
Summary
Leveraging human genetics, including diverse data types and techniques, enhances drug development success. A 5-step approach aids genetic target selection, despite challenges in data translation and variant identification.
Area of Science:
- Genetics
- Pharmacology
- Drug Discovery
Background:
- Drug development success rates can be improved by utilizing human genetics.
- Various genetic data types and techniques offer insights into human diseases.
Purpose of the Study:
- To present a comprehensive 5-step approach for selecting and validating genetic targets for drug development.
- To highlight the benefits of integrating diverse genetic evidence and discuss current challenges.
Main Methods:
- Utilizing genome-wide association studies, whole-exome sequencing, and whole-genome sequencing.
- Integrating evidence from Mendelian diseases, coding variants, and common variation.
- Employing human knockouts for reverse genetic screening and phenome-wide association studies for drug repurposing.
Main Results:
- Layering genetic evidence strengthens the case for a genetic target.
- Human knockouts enable identification of physiologically relevant targets.
- Phenome-wide association studies can reveal new therapeutic indications.
Conclusions:
- A structured 5-step approach optimizes genetic target selection for drug discovery.
- Challenges remain in genetic data translation, rare variant identification, and biobank integration.
- Human genetics provides a powerful foundation for developing effective medicines.
Keywords:
Geneticsdrug developmentfunctional genomicsgenome-wide association study (GWAS)genomicskidneyreviewtranslational scienceMore Related Videos
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