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CRISPR can treat common form of inherited blindness, early data hint

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A CRISPR therapy injected directly into the eye shows promise in treating the most common form of inherited vision loss in children, an early trial suggests.

This form of vision loss, called Leber congenital amaurosis (LCA), is often evident at birth and results from the dysfunction or death of light-sensing cells called photoreceptors in the retina, at the back of the eye. Such problems happen due to mutations in any of at least 20 genes. 

Some of the most common causes of LCA are mutations in the gene that codes for centrosomal protein 290 (CEP290). More than three-quarters of the people with the disease carry a particular mutation that affects CEP290, which is crucial for photoreceptors to function properly.

LCA currently has no cure — but now, there's evidence that the famous CRISPR gene-editing tool could be safely used to improve the vision of some people with the condition. The results of the early-stage trial were published May 6 in The New England Journal of Medicine

Related: CRISPR 'will provide cures for genetic diseases that were incurable before,' says renowned biochemist Virginijus Šikšnys

The results show the promise of using CRISPR to treat inherited eye diseases, Dr. Mark Pennesi, co-author of the report and a researcher at Oregon Health & Science University, told Live Science in an email. 

"This is just a start and more work is needed, but the proof of concept is exciting," he said. (Pennesi is a consultant with Editas Medicine, the trial's sponsor.)

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