Gene-editing drugs are moving from lab to clinic at lightning speed

The promising treatments still face technical and economic hurdles, though


  • by
  • 07 31, 2024
  • in Science & technology

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.

  • Source Gene-editing drugs are moving from lab to clinic at lightning speed
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