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Decades after it was first discovered that it could target DNA, we are beginning to see the first therapeutic approaches based on genetic modification. One problem these events have faced is security. Although we can make them specific to the gene we want to modify, the human genome is very large, and even rare DNA sequences can appear multiple times by accident.
As a result, all of the early gene editing techniques were prone to so-called off-target effects, in which they only change the wrong sequence. This may be a rare occurrence, but change enough cells – and treatment often requires many changes – and mistakes are inevitable.
Much effort has gone into finding ways to reduce or eliminate undocumented variables. In a recent issue of Nature, researchers described the evolution of the AI protein-folding software AlphaFold to help identify key regions of gene-editing proteins that cause dysfunction. Then the areas were changed to reduce the problems.
