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Gene therapy

Primed for take-off

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.

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Figure 1: Life-saving vectors.


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Correspondence to Philippe Leboulch.

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Leboulch, P. Primed for take-off. Nature 500, 280–282 (2013).

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