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Gene therapy

Primed for take-off

Nature volume 500, pages 280–282 (2013)Cite this article

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Gene therapy is finally getting a bumper crop of data that show clinical efficacy after fine-tuning of key parameters that control safety and potency. Supporting evidence comes from treatment of two life-threatening human diseases.

The development of the field of gene therapy shares many similarities with the history of aviation. Each is based on deceptively simple principles: the introduction of a therapeutic gene into cells and the flow of air over an aircraft's wing. However, the devil is in the details, and the details are in the numbers. Each field was marred by shortcomings and adverse events early on. But in spite of naysayers lacking vision, both fields continued their quest, and there is now firm hope that gene therapy will soon do for medicine what aeroplanes did for transportation. Good reason for this optimism comes from the results of two studies published in Science by Biffi et al.1 and Aiuti et al.2.

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Figure 1: Life-saving vectors.

References

  1. Biffi, A. et al. Science http://dx.doi.org/10.1126/science.1233158 (2013).

  2. Aiuti, A. et al. Science http://dx.doi.org/10.1126/science.1233151 (2013).

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  1. Philippe Leboulch is at the Institute of Emerging Diseases and Innovative Therapies, CEA, INSERM, University of Paris 11, 92265 Fontenay-aux-Roses, France. He is also at the Brigham & Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA.,

    Philippe Leboulch

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  1. Philippe Leboulch
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Correspondence to Philippe Leboulch.

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Leboulch, P. Primed for take-off. Nature 500, 280–282 (2013). https://doi.org/10.1038/500280a

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