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For kids with rare genetic disorders, customized CRISPR treatments offer hope
The gene-editing technique is effective for treating some illnesses but it's been too expensive to consider it for rare conditions. A new approach in the works could make it more widely available.
In principle, CRISPR, the gene-editing technique that enables scientists to easily make very precise changes in genes, could be a godsend for patients like Lucy. "When Lucy was diagnosed, I asked a bunch of my basic science friends who work at Genentech and all these other big companies in the Bay Area and I said, "Can't we just CRISPR this? One day, while out fundraising at a farmer's market, she bumped into Fyodor Urnov, who runs the Innovative Genomics Institute at the University of California, Berkeley.
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