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Ready to Go, But?

A number of CRISPR trials are poised to commence in people — and some already have — but Technology Review points out that researchers don't know whether the approach even works in non-human primates.

In Europe, CRISPR Therapeutics is seeking approval from regulators to test its beta thalassemia treatment and in the US, Editas Medicine has said it would be seeking its approval from regulators to test its congenital blindness therapy. Meanwhile, trials have pushed ahead in China testing whether CRISPR approaches are effective in treating cancer.

Tech Review writes, though, that there isn't much published data on CRISPR in monkeys to show that the gene-editing approach is safe and effective. It notes that the National Institutes of Health's Cynthia Dunbar and Hans-Peter Kiem at the Fred Hutchinson Cancer Research Center have each been studying gene editing using CRISPR in a monkey model of sickle cell anemia. They've found that the expected cuts do occur, but not always at a high rate.

Editas' Charles Albright tells Tech Review that they've used CRISPR in monkeys to correct the blindness mutations in about 10 percent of photoreceptor cells. While he adds that's not enough to restore full vision, it does allow them to see better.