in vivo therapy

In vivo therapy delivers the gene or the edit directly inside the body, so the change happens in the patient's own tissue where the cells live. The Latin in vivo means within the living body. Instead of sending cells out to a workshop, this is the house-call approach: the repair team comes to you and works on the cells right where they are, no removal required.

A treatment — such as a virus carrying a healthy gene, or editing tools packaged in tiny fat bubbles — is injected or infused and travels to the target cells, for example in the eye, muscle, or liver. There it slips its cargo inside, and the cells make the change in place. Nothing is taken out and grown in a dish; the patient's body is the workshop.

The appeal is simplicity: in principle one injection, no custom cell manufacturing. The challenge is aim and control. The therapy must reach enough of the right cells without straying to the wrong ones, the dose has to be right inside a living system, and because the change is made in place there is no dish step to screen out edits that missed their mark.