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Gene therapy's out-of-body experience

With an approval likely at the EMA, ex vivo gene therapy in hematopoietic stem cells appears poised for prime time.

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Figure 1: HSC gene therapy timeline.
Figure 2: In first-generation γ-retroviral vectors, the enhancer and promoters (E/P) of the LTRs can trans-activate cellular proto-oncogenes adjacent to the vector integration sites (dashed lines).

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Scott, C., DeFrancesco, L. Gene therapy's out-of-body experience. Nat Biotechnol 34, 600–607 (2016). https://doi.org/10.1038/nbt.3592

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