The most difficult thinking about gene therapy involves normally healthy people who have mutations that are highly predictive of a disease. Geneticists call this penetrance. It includes people who have the repeats of the motif CAG in a single gene and are almost certain to get Huntington’s disease, or those with mutations in the PDK gene family, which almost certainly cause polycystic kidney disease.
And so, there’s the rub. We’d surely accept some risk to edit the genes of a dangerously ill child; but do you edit motifs in genes that are merely likely to cause a disease?
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