CRISPR gives us the ability to correct genetic mutations, and given that such mutations are responsible for more than 6,000 human diseases, the tech has the potential to dramatically improve human health.
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Injecting a CRISPR therapy right into the bloodstream has been a problem, though, because the therapy has to find the right cells to edit… Now, researchers from Intellia Therapeutics and Regeneron Pharmaceuticals have demonstrated for the first time that a CRISPR therapy delivered into the bloodstream can travel to desired tissues to make edits.
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During a phase 1 clinical trial, Intellia researchers injected a CRISPR therapy dubbed NTLA-2001 into the bloodstreams of six people with a rare, potentially fatal genetic disorder called transthyretin amyloidosis… After just one injection of NTLA-2001, the three patients given a higher dose saw their levels of the protein drop by 80% to 96%.
“This is a wonderful day for the future of gene-editing as a medicine,” Fyodor Urnov, a UC Berkeley professor of genetics, who wasn’t involved in the trial, told NPR. “We as a species are watching this remarkable new show called: our gene-edited future.”
