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It is one thing to anticipate what gene therapy will look like in 5 years, let alone 10. The deeper we peer into the crystal ball, the less we can rely on what we see. So, let us put scrying and intuition aside. Instead, let us take a close look at gene therapy’s present state and how it has come to be. We will identify the forces behind gene therapy’s recent advances—as well as newly emergent forces—and we will keep in mind that these forces will likely wax and wane. Then we will be better able to predict what gene therapy will look like in the near future (or even the not-so-near future).

Vector improvements

Gene therapy can be divided into in vivo and ex vivo treatments. In the former, genetic material is delivered directly to the host by means of a vector such as a virus or nanoparticle. In cell-based gene therapies, on the other hand, genetic material is introduced to a patient’s cells ex vivo, and then the modified cells are returned to... see more