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Updated: Feb 13, 2026

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Published on: May 22, 2021
Translating pharmacogenetics and pharmacogenomics into drug development for clinical pediatrics and beyond
1Section of Developmental Pharmacology and Experimental Therapeutics, Children's Mercy Hospital, 2401 Gillham Road, Kansas City, MO 64108, USA. sleeder@cmh.edu
Insights
Pharmacogenetic and pharmacogenomic research in children is crucial due to rapid developmental changes. Understanding gene networks can prevent adverse drug reactions and inform novel pediatric treatments.
Area of Science:
- Pediatric pharmacology
- Developmental biology
- Genomics
Background:
- Human development is a dynamic process from conception to adolescence.
- Pharmacogenetic and pharmacogenomic data in children are lacking.
- Gene networks influence growth, development, and drug responses.
Purpose of the Study:
- To highlight the importance of pharmacogenetic and pharmacogenomic research in pediatric populations.
- To underscore the need to understand gene-drug interactions during development.
- To explore the potential for novel therapeutic strategies in childhood diseases.
Main Methods:
- Review of existing literature on pharmacogenetics and developmental biology.
- Analysis of gene networks involved in growth and development.
- Examination of potential impacts on drug-induced birth defects and pediatric adverse drug reactions.
Main Results:
- Child development significantly impacts drug response and susceptibility to adverse effects.
- Understanding gene networks can predict risks for drug-induced birth defects.
- Pharmacogenomics offers potential for personalized medicine in pediatrics.
Conclusions:
- Pharmacogenetic and pharmacogenomic studies in children are essential due to developmental changes.
- Research in this area can improve pediatric drug safety and efficacy.
- Investing in pediatric pharmacogenetics and pharmacogenomics research is critical for all age groups.
Abstract:
Pharmacogenetic and pharmacogenomic investigations conducted in children must consider that human development from conception through to adolescence is a rapidly changing, dynamic process. An improved understanding of the gene networks that are involved in growth and development and of the unintended consequences of modulating those systems could provide insights into the susceptibility of an individual to drug-induced birth defects and to pediatric adverse drug reactions. Furthermore, these technologies potentially present the opportunity to develop novel, effective treatments for childhood diseases and for adult diseases that manifest primarily during childhood. The lack of pharmacogenetic and pharmacogenomic investigations in children and the potential to impact on all age groups provides a considerable incentive to invest in this area of research.
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