Understanding how Cas9 is able to locate specific 20-base-pair target sequences within genomes that are millions to billions of base pairs long may enable improvements to gene targeting and genome editing efforts in bacteria and other types of cells.
I have always focused on basic research, motivated by a desire to understand the world.
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.
One of the problems in the biotech world is the lack of women in leadership roles, and I'd like to see that change by walking the walk.
In the past, when we've tried gene therapy, we haven't had tools that have allowed targeted gene correction.
I think that for a lot of women there's a subtle but unfortunately effective discouragement of women pursuing the STEM fields.
Is it more ethical to edit embryos or to screen a lot of embryos and throw them away? I don't know the answer.
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.
I was kind of a nerdy, geeky type. And I loved math. People teased me about it. I felt pretty much like an outcast.
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.
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.
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.
