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01 октября 2017, 00:30

Researchers Repair Human Embryos with “Chemical Surgery”

A team at Sun Yat-sen University in China has managed to use “chemical surgery” to rewrite parts of the genetic code of a human embryo. If this sounds familiar, you might be thinking of the CRISPR technique, but this is quite a bit different. Where CRISPR cuts out old code and splices in new code, the researchers performed direct edits to the genetic code.

The technique was first announced last year by a Harvard team. This is the first time it’s been used in human embryos, though they were not implanted after testing. More of a proof-of-concept, the group published their results in the journal Protein and Cell.

The goal was to find a potential cure for the rare blood disorder called thalassemia. This causes a reduction in hemoglobin, meaning that the body can’t carry enough oxygen. Red blood cells in patients with thalassemia also don’t tend to live quite as long or carry oxygen as efficiently, causing a myriad of chronic issues. Because the condition can be caused by a single mutation, researchers thought it’d be a good target for the new technique.

Also known as “base editing,” the technique works by partially splitting DNA section, and then flipping one of the base pairs. All DNA is made up of four nucleotides, which for the basic code. Adenine, Thymine, Guanine, and Cytosine exist as the A, T, G, and C of the code of life. Kinda like computer’s 0s and 1s (actually not at all, but it’s a useful-ish analogy).

Base editing, in its current form, can only switch one nucleotide at a time, and can only fix certain ones — G to an A, in this case. That’s limited in use, but it can fix tons of disorders which cause severe problems because of a single mutation. With this, we can flip the DNA back to healthy code, and without having to slice out whole chunks as one typically does with CRISPR. Plus, given recent misgivings about CRISPR, and specifically the inability to account for how cells implement new DNA, this could be a valuable new tool in medicine.

“Base editors directly perform chemical surgery on the target DNA base to convert one base pair to a different base pair (that is, to make a point mutation),” David Liu, lead on the Harvard team that invented the method, told Gizmodo. “Direct chemical surgery is an appropriate analogy because base editors actually catalyze the rearrangement of atoms on the target DNA base to transform that DNA base from a C to something that looks like a T (for example).”

Liu wasn’t involved in the study, but is understandably fascinated by it. “To my knowledge, the study is the first to use base editing to correct a pathogenic mutation in human embryos,” he said.

For now, the hope is that base editing could be used on parents to prevent their kids from having one of these disorders, but it may be possible to edit code in living patients too. That will actually probably hit the market sooner, just by nature of the fact that testing someone who is alive is usually a lot easier than editing code in a pre-baby and then waiting to see how that person’s health turns out decades down the line.

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Source: https://www.geek.com/science/researchers-repair-human-embryos-with-chemical-surgery-1717928/?source