My understanding of the gene therapy approach is basically “engineer a virus to deliver a good frataxin gene directly into the nerve cells.” Whereas I think genre editing is more like “take out some blood cells, edit their frataxin genes, put them back, then hope the blood cells glomp onto nerve cells and give them their frataxin.” (Someone correct me if I misunderstood the paper, my degree is not in biology)
So… It sounds like the first thing would be more effective? Maybe? But time and science will tell.
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