MIT Technology Review December 8, 2023
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The lucky break behind the first CRISPR treatment
The world’s first commercial gene-editing treatment is set to start changing the lives of people with sickle-cell disease. It’s called Casgevy, and it was approved last month in the UK. US approval is pending this week.
The treatment, which will be sold in the US by Vertex Pharmaceuticals, employs CRISPR, which can be easily programmed by scientists to cut DNA at precise locations they choose.
But where do you aim CRISPR, and how did the researchers know what DNA to change? That’s the lesser-known story of the sickle-cell breakthrough, which doesn’t rely on fixing the genes responsible for the mutation that leaves...
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