One can envisage taking cells from a patient with sickle-cell anaemia or an inherited blood disorder and using the Cas9 system to fix the underlying genetic cause of the disease by putting those cells back into the patient and allowing them to make copies of themselves to support the patient's blood.

As mechanistic biologists, we are hoping that by understanding how the virus works at the molecular level, we will be able to predict with more accuracy how it will evolve.

In the past, when we've tried gene therapy, we haven't had tools that have allowed targeted gene correction.

Jennifer Doudna Quotes