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.