Gene therapy for renal diseases

Y. H. Lien, L. W. Lai

Research output: Contribution to journalReview articlepeer-review

21 Scopus citations


Gene therapy is a promising therapeutic approach for a variety of renal diseases including both inherited and acquired diseases. In vivo gene transfer in the kidney using viral or non-viral vectors have been reported. These approaches have been tested in a few animal models of renal diseases, including experimental glomerulonephritis, ischemic renal failure, and carbonic anhydrase II deficiency. Selection of vectors, routes, and therapeutic genes is critical to the success of gene therapy targeted to the specific compartment of the kidney. Limitations of gene therapy for renal diseases exist and consist of: duration of transgene expression is short, transfection efficiency is not adequate, immune reactions are induced by adenoviral vector, and insertional mutagenesis may be caused by retroviral and adeno-associated vital vectors. Further studies are needed for improvement of gene delivery, minimization of side effects and development of cell-specific and long-term regulated gene expression.

Original languageEnglish (US)
Pages (from-to)S-85-S-88
JournalKidney International, Supplement
Issue number61
StatePublished - 1997


  • Cell-specific gene expression
  • Drug delivery
  • Gene therapy
  • Liposome-mediated gene therapy

ASJC Scopus subject areas

  • Nephrology


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