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Base Editing

2 GoKawiil briefs on this topic

Base editing used in human embryos reveals role of key early-development gene

Two independent research teams, led by Bower and colleagues and by Jerabek and colleagues, applied base editing—a precise form of genome editing—directly to human embryos to study how a specific gene shapes the earliest stages of development. Their experiments, published in Nature, show how embryos respond when particular DNA letters are altered, offering a clearer picture of this critical developmental window.

Base editing achieves near-complete PCSK9 correction in human embryos, but risks remain

Researchers delivered the ABE8e-V106W base editor as a protein into fertilized human embryos and successfully edited all copies of the PCSK9 gene without producing insertions or deletions, allowing embryos to develop to the blastocyst stage and yielding homozygous edited stem cell lines. However, they also observed occasional chromosome breaks, mosaic editing at nearby and off-target sites, and frequent embryo arrest when the editor was delivered as mRNA instead of protein.