TL;DR
Most gene-editing drugs change DNA, the permanent master copy in a cell. RNA editing goes after the working copy instead: the message a cell makes from a gene each time it uses it. Fix the message and the cell builds the right protein, while the DNA itself is never touched. The FDA has now agreed that one trial, with an early look at one year, could be enough to approve an RNA-editing drug from Wave Life Sciences. Nobody has yet shown that the drug works. But regulators are setting short, clear paths for editing drugs, and that could put a new kind of genetic repair in front of patients years sooner than the usual two-big-trials route would.
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