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Gene therapy for pediatric genetic kidney diseases

Yi Lu1,2, Yandong Song1, Shaokai Sun2

  • 1Key Laboratory of Immune Microenvironment and Disease (Ministry of Education) The Province and Ministry Co-sponsored Collaborative Innovation Center for Medical Epigenetics Department of Biochemistry and Molecular Biology School of Basic Medical Sciences Tianjin Institute of Urology The Second Hospital of Tianjin Medical University Tianjin Medical University Tianjin China.

Pediatric Discovery
|July 8, 2025
PubMed

Insights

Gene therapy offers a promising future for treating pediatric genetic kidney diseases, despite current limitations. This review explores gene therapy strategies, challenges, and potential solutions for conditions like Alport syndrome and polycystic kidney disease.

Area of Science:

  • Pediatric Nephrology
  • Genetics
  • Molecular Medicine

Background:

  • Genetic kidney diseases are a primary cause of chronic kidney disease in children.
  • Pathogenic genes are identified, but disease mechanisms and treatments remain limited.
  • Gene therapy is successful for other genetic disorders but lags in kidney disease applications.

Purpose of the Study:

  • To review gene therapy strategies for pediatric genetic kidney diseases.
  • To provide an overview of current treatments and emerging gene therapy research.
  • To discuss challenges and solutions for gene therapy in pediatric kidney disorders.

Main Methods:

  • Comprehensive literature review of gene therapy strategies.
  • Analysis of current treatment options for genetic kidney diseases.
  • Focus on gene therapy research for Alport syndrome and polycystic kidney disease.

Main Results:

  • Gene therapy is a viable approach for genetic kidney diseases.
  • Significant research is ongoing for Alport syndrome and polycystic kidney disease.
  • Challenges in delivery and efficacy need to be addressed.

Conclusions:

  • Gene therapy holds promise for treating pediatric genetic kidney diseases.
  • Further research is needed to overcome challenges and develop effective treatments.
  • Focus on specific conditions like Alport syndrome and polycystic kidney disease is crucial.
Keywords:
AAVAlport syndromeCRISPR‐Cas9LNPPKDgene therapypediatric genetic kidney diseases

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