American - Scientist | -
Is it more ethical to edit embryos or to screen a lot of embryos and throw them away? I don't know the answer.
Jennifer Doudna
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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.
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In the past, when we've tried gene therapy, we haven't had tools that have allowed targeted gene correction.
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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.
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The idea that you would affect evolution is a very profound thing.
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The more we know, the more we realize there is to know.
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I was kind of a nerdy, geeky type. And I loved math. People teased me about it. I felt pretty much like an outcast.
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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.
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People get comfortable with technologies.
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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.
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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.
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I have always focused on basic research, motivated by a desire to understand the world.
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