Since its debut in 2012, CRISPR gene editing has held the promise of curing most of the over 6,000 known genetic diseases. Now it’s being put to the test.
In the first spate of clinical trials, scientists are using CRISPR/Cas9 to combat cancer and blood disorders in people. In these tests, researchers remove some of a person’s cells, edit the DNA and then inject the cells back in, now hopefully armed to fight disease. Researchers are also set to see how CRISPR/Cas9 works inside the human body. In an upcoming trial, people with an inherited blindness will have the molecular scissors injected into their eyes.
Those tests, if successful, could spur future trials for Duchenne muscular dystrophy, cystic fibrosis and a wide variety of other genetic diseases, affecting millions of people worldwide.
Even if these first trials don’t pan out as hoped, CRISPR won’t be shelved, Albright thinks. “This is a technology that’s here to stay,” he says. “If this doesn’t work, it’s going to be more about the underlying biology or our ability to deliver the editing machinery.”
There’s precedence that perseverance — and choosing the right disease to target — can eventually pay off.
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