Applications of gene therapy to kidney disease

Basil Hanss, Leslie A. Bruggeman

Research output: Contribution to journalReview articlepeer-review

9 Scopus citations


Purpose of review: This review summarizes recent applications of somatic cell gene therapy to the treatment of monogenetic renal diseases, renal cell carcinoma, and for the induction of tolerance in solid organ transplantation. In addition, several new gene therapy techniques will be discussed including gene and messenger RNA repair strategies, as well as methods designed to modify the expression of normal genes that may have application in the treatment of multigenetic disorders. Recent findings: Animal studies have demonstrated prolonged graft survival after the successful induction of tolerance to alloantigens via hemalopoietic molecular chimerism. Ongoing clinical trials for renal cell carcinoma are encouraging, in that IL-2 gene therapy using non-viral vector systems can reduce the tumor burden. However, limited progress has been made towards applying gene therapy for the most common genetic disorders of the kidney, autosomal dominant polycystic kidney disease and Alport syndrome. Basic research on novel gene repair and expression modulation techniques provide additional gene therapy options for the treatment of viral infections such as HIV-1 and monogenetic disorders. Summary: Gene therapy holds enormous potential for the treatment of genetic and acquired diseases. Current pre-clinical studies and clinical trials provide encouraging results that gene therapy can become a useful treatment option. However, before gene therapy has widespread application, technical progress must be made in all aspects of treatment design, including optimizing vector and delivery systems and the ability to modify long-term cell populations such as stem cells.

Original languageEnglish
Pages (from-to)439-445
Number of pages7
JournalCurrent Opinion in Nephrology and Hypertension
Issue number4
StatePublished - Jul 2003


  • Alport syndrome
  • Genetic disorders
  • Polycystic kidney disease
  • Renal cell carcinoma
  • Transplantation


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