Gene-editing drugs are moving from lab to clinic at lightning speed
The promising treatments still face technical and economic hurdles, though
One autumn day in 2020 Patrick Doherty was walking his dog up a steep mountain in County Donegal, Ireland, when he noticed he was, unusually for him, running out of breath. The eventual diagnosis was terrifying: amyloidosis, a rare genetic disease that caused a protein, amyloid, to build up in his organs and tissues. The prognosis was even worse: it would cause him years of pain until it finally killed him. In the face of such terrible fortune, though, Mr Doherty had a stroke of luck. He was able to join a trial of a new medical therapy and, with just a single injection, was apparently cured. Now, he continues to walk his dog up that steep mountain in County Donegal every week.
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