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Homologous Recombination Based Gene Therapy
Li-Wen Lai, Yeong-Hau H. Lien

Department of Medicine, Sections of Endocrinology and Nephrology, University of Arizona Health Sciences Center, Tucson, Ariz., USA

Address of Corresponding Author

Exp Nephrol 1999;7:11-14 (DOI: 10.1159/000020578)

  Key Words

• Homologous recombination
• Gene targeting
• Gene therapy

  Abstract

Background/Aims: Most of the current expression vector based gene therapy protocols fail to achieve clinically significant transgene expression required for treating genetic diseases. Homologous recombination, initially considered to be of limited use for gene therapy because of its low frequency in mammalian cells, has recently emerged as a potential strategy for developing gene therapy. Methods: Six recent studies of homologous recombination in mammalian cells are reviewed. Different approaches have been used in these studies including RNA/DNA chimeric oligonucleotides, small or large homologous DNA fragments, or adeno-associated viral vectors. Results: Most of these studies show a reasonable frequency of homologous recombination which warrants further in vivo testing. Conclusions: Homologous recombination based gene therapy has the potential to develop into a powerful therapeutic modality for genetic diseases. It can offer permanent expression and normal regulation of corrected genes in appropriate cells or organs and probably can be used for treating dominantly inherited diseases such as polycystic kidney disease.

  Author Contacts

Li-Wen Lai, PhD
Department of Medicine, Section of Endocrinology
University of Arizona Health Sciences Center
Tucson, AZ 85724 (USA)
Tel. +1 (520) 626 0972, Fax +1 (520) 626 2024, E-Mail llai@u.arizona.edu

  Article Information

Received: Received: May 5, 1998
Accepted: May 28, 1998
Number of Print Pages : 4
Number of Figures : 1, Number of Tables : 1, Number of References : 15