On Friday, the Food and Drug Administration approved the first-ever drug to rely on a Nobel-prize-winning technique that mutes disease-causing genes — a watershed moment for that field of research, and a starting pistol for the race to find a way to use the therapy for other debilitating genetic diseases like ALS or Huntington’s.
But already, a major question looms: What if the technique won’t work in other parts of the body?
The first-ever RNAi drug, patisiran, targets the liver. So, too, do similar, already-approved therapies in the broader category of “sequence-based drugs.” There’s a simple reason for that: The liver is an easy target. The liver is the body’s filter; if something is in your blood, it will eventually pass into the organ.
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