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Beginning of the End?

With recent advances in genome editing, Jennifer Doudna from the University of California, Berkeley, says the end may be nigh for genetic diseases.

Doudna recently received, along with Emmanuelle Charpentier from the Helmholtz Center for Infection Research, the life sciences Breakthrough prize for their work on CRISPR/Cas9. As Technology Review has noted, there are others vying for ownership of the approach.

In a Q&A with the World Economic Forum's Katia Moskvitch, Doudna says that Cas9 can help re-write the genome.

"You can think about it like a computer code," she says. The DNA of the cell is analogous to the code that programs a computer. Imagine that you try to run the code, and there's an error in it – then the computer doesn't run very well. It's the same in the cell of an organism. If there's a mutation in the DNA, it affects the cell's ability to grow and function normally."

Editing techniques like CRISPR/Cas9 — which she says is easier to use than zinc-finger nucleases or TALENs — can be used to fix mutations that lead to disease.

Doudna adds that she's set up a lab that's focusing on developing this approach to treat sickle cell anemia, cataracts, and more.

In a separate interview with the Huffington Post at Davos, Doudna laments that women interested in the sciences can sometimes be discouraged from pursuing that path.

"Women have natural curiosities, as do men, and we just want to show women they can pursue these fields and they can be feminine, they can be mothers, they can be wives, they really can do all those things and do it on their own terms," Doudna adds.

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