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Ligand-directed targeting of genes to the site of disease

Nature Medicine volume 9, pages 135–139 (2003)Cite this article

Fundamental changes in the understanding of the primary influences that govern specific gene delivery, combined with rational approaches to engineer the gene transfer vectors, are now transforming targeted in vivo gene transfer from concept to reality. Many viral-based vectors have been designed to avoid gene transfer through their native receptors and redirected to a variety of tissue- and tumor-specific receptors. Non-viral vectors have likewise been engineered to avoid nonspecific gene transfer. Future challenges include advancing these vectors into clinical testing, designing improvements to avoid innate and acquired immunity, and elucidating the mechanisms that govern their biodistribution and pharmacokinetics.

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Figure 1: One of the ultimate goals of targeted gene transfer is to engineer the vectors so that they can be administered through the circulation.

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Acknowledgements

The author thanks C. Richter King for his critical review of this manuscript.

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    Thomas J. Wickham

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Wickham, T. Ligand-directed targeting of genes to the site of disease. Nat Med 9, 135–139 (2003). https://doi.org/10.1038/nm0103-135

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