CRISPR milestone: Crippling transthyretin amyloidosis disease now treatable through gene editing, paving way for addressing other genetic diseases

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Credit: Shutterstock
Credit: Shutterstock

[65-year-old Patrick] 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.

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“It’s terrible prognosis,” Doherty says. “This is a condition that deteriorates very rapidly. It’s just dreadful.”

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.

“I thought: Fantastic. I jumped at the opportunity,” Doherty says.

On [June 26], 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.”

The advance is being hailed not just for amyloidosis patients but also as a proof-of-concept that CRISPR could be used to treat many other, much more common diseases. 

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