The ability to edit the genes of human beings by changing the code of life – DNA – has dramatically improved over the last decade. Current tools, likeand , are extremely powerful but have traditionally suffered from a lack of precision, high error rate and limited scope, hampering their ability to treat human genetic disease. A breakthrough gene-editing tool, developed by a team of researchers at Harvard University and unveiled Monday, has the ability to make extremely precise DNA edits, ushering in a new era of genetic manipulation.
“In many respects this first report is the beginning rather than the end of a longstanding aspiration … to be able to make any DNA change in any position of a living cell or organism including, potentially, human patients with genetic diseases,” says David Liu, a chemist at Harvard’s Broad Institute and co-author of the new study.
Gene editing involves changing the four bases of the DNA code, designated by the letters A, C, T and G, by deleting them, inserting them, modifying them or a combination of the three.
The research, published in the journal Nature on Oct. 21 and led by Andrew Anzalone, describes the new technique, dubbed “prime editing,” in a series of elegant experiments using four human cell lines and mouse brain cells. Performing 175 different DNA edits, the researchers show prime editing can change DNA with incredible precision and, importantly, introduces errors at a much lower rate than previous gene-editing technologies. To demonstrate this, the team corrected the genetic mutations for two human genetic diseases, sickle cell and Tay-Sachs disease, in human cells. They showed the necessary edits could be made to the DNA to reverse the genetic mutations causing those diseases.
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