![](https://www.rickilewis.com/rails/active_storage/representations/redirect/eyJfcmFpbHMiOnsibWVzc2FnZSI6IkJBaHBBN2VSQXc9PSIsImV4cCI6bnVsbCwicHVyIjoiYmxvYl9pZCJ9fQ==--660afc7c867826f3da6f74f47b667e091192b0e4/eyJfcmFpbHMiOnsibWVzc2FnZSI6IkJBaDdCem9MWm05eWJXRjBTU0lJYW5CbkJqb0dSVlE2RkhKbGMybDZaVjkwYjE5c2FXMXBkRnNIYVFMZ0FXa0M0QUU9IiwiZXhwIjpudWxsLCJwdXIiOiJ2YXJpYXRpb24ifX0=--d00c0b801be2eac628730b2b4ffb891cbdd69dfe/Tratamiento-epidermolisis-bullosa.jpg)
The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn't a one-and-done.
My now decade-old book The Forever Fix: Gene Therapy and the Boy who Saved It, told the stories of children who had received one-time deliveries of working copies of genes, to compensate for their mutations. The initial gene therapies helped people with a form of inherited retinal blindness to see and children with profound immune deficiencies to survive. Today, several single-gene blood, brain, muscle, and metabolic disorders are responding to one-time infusions of a gene therapy.
The biology behind a single-gene condition suggests how a particular gene therapy would be delivered, targeted, and the effect maintained. Compared to slash-and-burn technologies like standard chemo and radiation that impact cells beyond the targeted ones, a gene therapy is both rational and tailored.
To continue reading, go to DNA Science, where this post first appeared.