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Integration errors aren’t the main concern with treating patients - it’s the off target effects. In other words, changes to genes you didn’t expect. You don’t w
by mbreese 7y ago
Integration errors aren’t the main concern with treating patients - it’s the off target effects. In other words, changes to genes you didn’t expect. You don’t want to fix one problem, only to cause something else.
IIRC, the reason why the integration errors were such a big deal is because they made it to the germline of the animal and could be transferred to offspring. This would have been extensively validated given the amount of press and regulatory pressure.