The dangers of editing the human genome are well-known: the power to change small aspects of people, tweaking them to our liking, means that even a mostly fruitless attempt to edit human embryos sparked widespread fear and criticism, with (mostly premature) speculation that designer babies could be right around the corner.
But the potential to use these same technologies to cure intractable diseases is huge.
In a recent essay for the New England Journal of Medicine, Eric S. Lander, the founding director of The Broad Institute and a professor at Harvard Medical School and MIT, clearly expresses the importance of not being hasty or foolish as this transformative research moves forward.
“It has been only about a decade since we first read the human genome,” he writes. “We should exercise great caution before we begin to rewrite it.”
But he also notes that while more discussion and regulation is necessary before these tools become a free-for-all, “genome editing also holds great therapeutic promise.”
Specifically, he mentions five diseases that could be completely eliminated with gene editing technology (once that technology becomes much more accurate and reliable than it is today):
- HIV. “Physicians might edit a patient’s immune cells to delete the CCR5 gene, conferring the resistance to HIV carried by the 1% of the U.S. population lacking functional copies of this gene,” he writes.
- Some forms of genetic blindness. Inactivating a certain variant of a gene in the retinal cells of the eye could stop some types of inherited, progressive blindness in their tracks, Lander suggests.
- Familial hypercholesterolemia. An inherited condition, familial hypercholesterolemia can lead to extremely high levels of “bad” cholesterol and heart attacks at a young age. Editing liver cells could fix this inherited disorder, Lander suggests.
- Sickle cell anemia. Lander suggests that editing blood stem cells could cure this disease, which affects about 100,000 Americans and can cause lifelong pain and even organ damage.
- Hemophilia. Another blood disorder that Lander says could be cured by editing blood stem cells, hemophilia causes frequent bruising, pain, and excessive bleeding due to missing or low levels of the proteins needed to create clots. About 1 in 5,000 babies in the US are born with classic hemophilia.
Unlike the kind of embryo editing that could lead to permanent changes in the human race, these edits would be made in babies, children, or adults. That means, Lander writes, “they pose no unique ethical issues because they affect only a patient’s own [ordinary] cells.”
Still, Lander points to “serious technical challenges” that would have to be overcome before any of these potential cures could be implemented. The technology is still a long way off from making these kinds of treatments a reality.
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