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The first medicine based on gene editing, a one-time therapy for sickle cell disease, was just approved in the United States. It’s a big moment for patients with sickle cell disease and for the technology called CRISPR, which powers the therapy.
I am Carolyn Johnson, a science reporter at The Washington Post, and I’ve been following the speedy trajectory of CRISPR from a scientific breakthrough in 2012 to a medicine that can alleviate human suffering 11 years later. On Tuesday, I answered your questions about the potential of this technology to transform medicine — and the challenges associated with it.
First, a quick primer: CRISPR is often compared to a pair of “molecular scissors” that can make targeted cuts in DNA, giving scientists the ability to easily and precisely alter the genome. Scientists often hear from families afflicted by genetic diseases hoping that this technology will help save their loved ones. It has also spurred controversies — including battles over who invented CRISPR and the fear that it will be used to create “designer babies.”
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