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Curr Opin Nephrol Hypertens. 2003 Jul;12(4):439-45.

Applications of gene therapy to kidney disease.

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Division of Nephrology, Mount Sinai School of Medicine, New York, New York, USA.



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.


Animal studies have demonstrated prolonged graft survival after the successful induction of tolerance to alloantigens via hematopoietic 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.


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.

[Indexed for MEDLINE]

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