He Inherited a Devastating Disease -- A CRISPR Gene-Editing Breakthrough Stopped It

upstart writes:

He Inherited A Devastating Disease. A CRISPR Gene-Editing Breakthrough Stopped It:

Patrick Doherty had always been very active. He trekked the Himalayas and hiked trails in Spain.

But about a year and a half ago, he noticed pins and needles in his fingers and toes. His feet got cold. And then he started getting out of breath any time he walked his dog up the hills of County Donegal in Ireland where he lives.

[...] Doherty found out he had a rare, but devastating inherited disease - known as transthyretin amyloidosis - that had killed his father. A misshapen protein was building up in his body, destroying important tissues, such as nerves in his hands and feet and his heart.

[...] So Doherty was thrilled when he found out that doctors were testing a new way to try to treat amyloidosis. The approach used a revolutionary gene-editing technique called CRISPR, which allows scientists to make very precise changes in DNA.

[...] On Saturday, researchers reported the first data indicating that the experimental treatment worked, causing levels of the destructive protein to plummet in Doherty's body and the bodies of five other patients treated with the approach.

"I feel fantastic," Doherty says. "It's just phenomenal."

[...] "This is the first example in which CRISPR-Cas9 is injected directly into the bloodstream - in other words systemic administration - where we use it as a way to reach a tissue that's far away from the site of injection and very specifically use it to edit disease-causing genes," says John Leonard, the CEO of Intellia Therapeutics, which is sponsoring the study.

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