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Receptor targeting of adeno-associated virus vectors

Gene Therapy volume 10, pages 1142–1151 (2003)Cite this article

Abstract

Adeno-associated virus (AAV) is a promising vector for human somatic gene therapy. However, its broad host range is a disadvantage for in vivo gene therapy, because it does not allow the selective tissue- or organ-restricted transduction required to enhance the safety and efficiency of the gene transfer. Therefore, increasing efforts are being made to target AAV-2-based vectors to specific receptors. The studies summarized in this review show that it is possible to target AAV-2 to a specific cell. So far, the most promising approach is the genetic modification of the viral capsid. However, the currently available AAV-2 targeting vectors need to be improved with regard to the elimination of the wild-type AAV-2 tropism and the improvement of infectious titers. The creation of highly efficient AAV-2 targeting vectors will also require a better understanding of the transmembrane and intracellular processing of this virus.

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Acknowledgements

This work was supported by the Deutsche Forschungsgemeinschaft (SFB 455), the Wilhelm Sander-Stiftung and the Bayerische Forschungsstiftung. We thank all members of the laboratory for many inspiring discussions and help during the work presented in this review, and Dr Susan King for critically reading the manuscript. The authors apologize to investigators whose work was not cited owing to limited space.

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  1. Genzentrum Ludwig-Maximilians-Universität München, München, Germany

    H Büning, M U Ried, L Perabo, F M Gerner, N A Huttner, J Enssle & M Hallek

  2. Medizinische Klinik III, Klinikum der Universität München, Groβhadern, München, Germany

    M Hallek

  3. GSF, National Research Center for Environment & Health, München, Germany

    M Hallek

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Büning, H., Ried, M., Perabo, L. et al. Receptor targeting of adeno-associated virus vectors. Gene Ther 10, 1142–1151 (2003). https://doi.org/10.1038/sj.gt.3301976

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