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Gene modification: Exploring the potential in treating kidney diseases

Pharmacological ResearchResearch Authors: Ubong S. Ekperikpe, Serena Zhao, Ilse S. DaehnAIIM Authors: Anisha Singla and Madison SchanzApproved by President Reda RiffiPublication Date: 2/1/2026

Comprehensive Summary

Ekperikpe et al. present a review which explores the potential of gene modification technologies as future treatments for chronic kidney disease (CKD).The authors reviewed recent advances in genetic and genomic technologies, including gene editing and delivery strategies, and examined their mechanisms, current applications, and limitations in treating CKD. Gene therapies have transformed treatments in other disease areas, but none are yet approved for kidney diseases. Emerging gene modification tools (e.g., CRISPR-based techniques) show promise in targeting genetic contributors to CKD and could enable precision editing to slow or reverse disease progression. The review also highlights the challenges of delivery methods, safety concerns, and adverse effects that must be addressed. The authors suggest that as understanding of CKD genetics and editing technologies improves, gene therapies may become viable in the future, but significant research is still required before clinical adoption.

Outcomes and Implications

This review is important because chronic kidney disease remains a major global health burden with limited options to halt or reverse disease progression. Gene modification strategies could shift treatment from symptom management to directly targeting underlying genetic causes of kidney damage. Clinically, advances such as CRISPR-based editing and improved gene delivery systems may eventually enable precision therapies for inherited or progressive kidney disorders. However, the authors emphasize that significant challenges—including safe delivery to kidney tissue, long-term safety, and regulatory approval—mean that widespread clinical implementation is still years away and will require further preclinical and clinical trials.

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