A new way to make powerful changes at will to the DNA of humans, other animals and plants, much like how a writer changes words in a story, could usher in a transformation in genetic medicine.
Scientists are not just excited about this recently discovered technique because it can snip and edit DNA with precision. It can also do the job more easily and cheaply than other gene-editing methods, making possible research that has historically been difficult, experts say.
Now some of the biologists who unlocked this tool, derived from the immune system of bacteria, are forming companies around it. Although this molecular system, known as Crispr, is not fully understood, researchers believe it can be harnessed to create therapies for intractable genetic diseases.
“In principle, this is a technology that could enable correction of genetic mutations that would otherwise lead to disease,” said Doudna, a professor of chemistry and biochemistry and molecular biology, in a telephone interview. She was among several experts who spoke at a UC Berkeley conference on the subject last month.
Ethical concerns
But because the method is in its infancy and has little precedent with the agencies that regulate medicines, it will almost certainly be a long time before a Crispr-based therapy makes it to market.
Its potential risks also concern some bioethicists.
The Latest on: Editing DNA
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The Latest on: Editing DNA
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The big deal between Regeneron and a Peninsula biotech co-founded by Jennifer Doudna is also a small deal. And that’s a huge deal.
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