“Regulators last year approved the world’s first medicine using Crispr, the Nobel Prize-winning tool for modifying genes. The medicine, for sickle-cell disease, a group of inherited blood disorders, involves extracting cells, editing them in a lab and putting them back in the patient’s body.
The experimental therapies Odunsi and others are receiving, by contrast, edit their cells inside their bodies. ‘In vivo’ gene editing, as the approach is called, could transform medicine. Several of the therapies are for cardiovascular disease, and if proven safe and effective could reach millions of patients.”
From Wall Street Journal.