Two clinics sit adjacent to one another in a new hospital in a medium-sized city in the developed world.
In one, a family waits for a long-scheduled appointment. Their daughter suffers from a rare inherited disease. One of her genes encodes for a protein that doesn’t perform its functions normally, and her degenerative ailment is most likely fatal. Their doctor is about to discuss a procedure that will remove cells from the affected organ, correct the inherited faults and reinfuse her cells, allowing her organ to perform its functions without the inborn errors that she and her family have been managing since her birth.
The procedure is expensive, requiring a payment plan over a decade financed by a start-up, and it will need chemotherapy and a hospital stay. But it’s a one-time fix, and if side-effects appear down the line, doctors will study them.
In another waiting room, a soon-to-be-expectant couple waits to meet a geneticist. The doctor will present a plan for their unborn baby: to alter a few lines of its DNA to reverse a rare disorder before it even starts and prevent it from being passed to their grandchildren. Both parents carry the recessive gene that causes the disease, but because of the opportunity to correct it after fertilisation, they decide to try to get pregnant. It’s supercharged IVF.
Both of these scenarios are cutting-edge and experimental.
The first is being tried around the world and has seen some breathtaking achievements. In the United States, more than two dozen such gene-editing therapies have been approved to tackle blindness, rare immune and genetic disorders, and some cancers. But they remain expensive and tailored to the very, very few.
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The second has been condemned. In-embryo edits – changes that would be replicated into reproductive cells and passed to future generations – are banned by many governments.
But either, or both, could become more common in the coming years. In March, scientists, genetics entrepreneurs, ethicists and policymakers pondered the future of the sector at the third International Summit of Human Genome Editing at the Royal Society in London.
It was on the same stage that a scientific scandal hit global headlines in 2018 when Chinese geneticist He Jiankui announced he had altered the DNA of the embryos of twin girls to protect them from an HIV infection in the future. He brought the embryos to term, was called “Dr Frankenstein” by some and sentenced to three years in prison by a Chinese court.
His revelation served as a curtain-raiser to a new era in which the codes that make our bodies can be edited. Work to correct genetic diseases has continued bounding forward.