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United StatesBusiness13 days ago

New Embryo Editing Technique Takes Us a Step Closer to Designing Babies Without Disease

Columbia University scientists have developed a new gene-editing technique using base editing to correct DNA errors in human embryos without causing chromosomal abnormalities. The study focused on repairing mutations in the PCSK9 gene, linked to high cholesterol, and the HBG genes, associated with blood disorders like sickle cell disease and thalassemia. While the method showed promise, it was not fully effective in all cells of the embryos.

Gene-editing human embryos—the sci-fi scenario that many have feared and many others have cheered—may now be a reality. Columbia University scientists say they have found an " efficient and precise " way to edit human embryos. Unlike earlier methods using CRISPR alone, this method works without introducing chromosomal abnormalities into the embryo or deleting large sequences of DNA.

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In their study, the scientists used a technique called base editing to repair "DNA nicks and mismatches" in human embryos, according to a preprint study published on June 1.

The process could allow scientists to repair embryonic DNA that might otherwise result in disease.

In the new study, Dieter Egli and colleagues—which include Nathan Treff of the DNA-testing startup Nucleus Genomics—focused on the PCSK9 gene, which regulates cholesterol, and the HBG genes, which control fetal hemoglobin production. Mutations in the PCSK9 gene can lead to high LDL cholesterol. Some think changes to the HBG genes could prevent sickle cell disease and thalassemia. The scientists inserted their base editors into early stage embryos with an eye toward altering these genes.

It wasn't perfect. In many cases, some cells in an embryo were successfully edited but not all of them, creating what are known as "mosaics."

But the genes they wanted to change were changed—without the sort of damage seen in the earlier technique.

"We're not saying this is going to be used tomorrow in the clinics," lead study author Dieter Egli told The New York Times . Even their paper has not yet been peer-reviewed.

Still, the results are already being heralded as promising and " impressive ."

They're a big improvement on earlier techniques, such as using CRISPR alone. In 2020, Egli and colleagues tried using this on human embryos to snip out a mutation that could cause blindness. But after that, the embryo repaired the removed gene effectively only about half of the time. Other times, the embryo would delete long sequences of DNA or destroy the entire chromosome where the gene in question was located. "It had absolutely catastrophic consequences," Egli told the Times .

The newer technique, developed by David Liu of Harvard and MIT's Broad Institute, combines a CRISPR molecule with other compounds to do something called base editing. This technique "allows scientists to make precise, single-letter changes to DNA," as Nature describes it.

Base editing has already been used to repair a rare genetic disorder in a baby .

The new experiment from Egli and his colleagues represent the first time this technique has been used to edit embryonic cells. Eventually, "base-editing technology could one day be used to help parents avoid discarding embryos in the IVF process," notes The Wall Street Journal .

Theoretically, this same technology could be used to do much more than repair abnormalities and damage: It could bring us one step closer to the "designer babies" constantly invoked by critics. The Nature , Times , and Journal articles all contain quotes from people worried about the ethical implications of embryonic gene editing or the potential for bad outcomes if it is used prematurely.

While the safety concerns may have merit for now, the other fears seem woefully misguided and overblown. We know that things like intelligence, personality traits, and athletic ability are the result of many genes working together. The dystopian scenario so many conjure when it comes to this stuff—rich people creating a race of superbabies who exacerbate inequality—is more of a sci-fi trope than a possible future of this technology.

Besides: We're on the cusp of developing technology that could save kids from horrible diseases! And could help more people realize their dreams of having children.

"Designer babies" conjures the idea of elitist and frivolous uses of this technology. But as this study suggests, we could also "design" babies to avoid heart disease or sickle cell anemia. That's something to celebrate.

Followup

On nationalizing AI: Last week, this newsletter covered a proposal by Sen. Bernie Sanders (I–Vt.) for the government to confiscate AI company stock. A few days later, President Donald Trump called for something similar.

"Leaders of 'all the big' AI companies are coming to the White House as early as next week to discuss the idea of the government taking stakes in the firms, Trump told reporters in remarks on Air Force One," reports The Washington Post . "The suggestion follows previous deals in which the Trump administration has taken stakes in major companies including chipmaker Intel, breaching traditions that previously protected American firms from government intervention."

Aligning government interests and AI company interests all but assures that the public interest will not be served, as critics of AI nationalization poi…

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Source document: Preprint Study Published on June 1

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ReasonIndependentCenter13 days ago
New Embryo Editing Technique Takes Us a Step Closer to Designing Babies Without Disease

Columbia University scientists have developed a new gene-editing technique using base editing to correct DNA errors in human embryos without causing chromosomal abnormalities. The study focused on repairing mutations in the PCSK9 gene, linked to high cholesterol, and the HBG genes, associated with blood disorders like sickle cell disease and thalassemia. While the method showed promise, it was not fully effective in all cells of the embryos.

Bias read (Center): The article presents scientific findings neutrally, focusing on the technical aspects of the research without taking a stance on ethical or political implications. It does not favor any ideological perspective and provides factual information about the study's methodology and results.

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  • studyPreprint Study Published on June 1