Scientists successfully used CRISPR to fix a mutation that causes disease. This is huge. – Eliza Barclay Aug 2, 2017, 3:50pm EDT


These embryos were made in the lab with sperm carrying a genetic mutation known to cause hypertrophic cardiomyopathy. CRISPR/Cas9 was used to correct the mutation.
OHSU

CRISPR/Cas9 is a gene editing technology that’s revolutionizing science at a breathtaking pace.

One of its most exciting, taboo, and controversial applications is tweaking the genes of eggs, sperm, or early embryos to alter a human life. This could one day mean the ability to create smarter or more athletic humans (yes, “designer babies”), but also the chance to knock out disease-causing genetic mutations that parents pass on to their children. We’re talking about eliminating mutations linked to diseases like breast and ovarian cancers or cystic fibrosis.

On Wednesday, a team of scientists reported that they have made major progress toward proving the latter is possible.

In a paper published in the prestigious journal Nature, a team led by Shoukhrat Mitalipov of Oregon Health and Science University described how it used CRISPR/Cas9 to correct a genetic mutation that’s linked to a heart disorder called hypertrophic cardiomyopathy in human embryos. And they did it without the errors that have plagued previous attempts to edit human embryos with CRISPR.

To be clear, the new work from OHSU was an experiment — the point was to test a concept, and the embryos used were never implanted into a woman’s uterus.

But the researchers were ultimately able to show that CRISPR/Cas9 can do what they hoped it would do. It cut the mutant gene sequence, prompted the embryos to repair the DNA with healthy copies of the gene, and eliminated the disease-causing mutation altogether from many of the embryos.

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