This Company Wants to Rewrite the Future of Genetic Disease—Without Crispr Gene Editing

Good News Notes:

CRISPR’S POTENTIAL FOR curing inherited disease has made headlines, including at WIRED, for years. ( Hereherehere, and here.) Finally, at least for one family, the gene-editing technology is turning out to deliver more hope than hype. A year after 34-year-old Victoria Gray received an infusion of billions of Crispr’d cells, NPR reported last week that those cells were still alive and alleviating the complications of her sickle cell disease. Researchers say it’s still too soon to call it a cure. But as the first person with a genetic disorder to be successfully treated with Crispr in the US, it’s a huge milestone. And with dozens more clinical trials currently in progress, Crispr is just getting started.

Yet for all its DNA-snipping precision, Crispr is best at breaking DNA. In Gray’s case, the gene editor built by Crispr Therapeutics intentionally crippled a regulatory gene in her bone marrow cells, boosting production of a dormant, fetal form of hemoglobin, and overcoming a mutation that leads to poor production of the adult form of the oxygen-carrying molecule. It’s a clever way around Crispr’s limitations. But it won’t work for a lot of other inherited conditions. If you want to replace a faulty gene with a healthy one, you need a different tool. And if you need to insert a lot of DNA, well, you’re kind of out of luck.”

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