In the past, when we've tried gene therapy, we haven't had tools that have allowed targeted gene correction.
There's already a lot of active research going on using the Crispr technology to fix diseases like Duchenne muscular dystrophy or cystic fibrosis or Huntington's disease. They're all diseases that have known genetic causes, and we now have the technology that can repair those mutations to provide, we hope, patients with a normal life.
We found that CAS9 has the ability to make a double-stranded break in DNA at sites that are programmed by a small RNA molecule. What was so important was that we could really show how the CAS9 protein worked.
The impression sometimes created among the public is that scientists are working away in their labs, and maybe they're not always thinking about the implications of their work. But we are.
I was kind of a nerdy, geeky type. And I loved math. People teased me about it. I felt pretty much like an outcast.
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.
