New Gene-Editing ‘Pencil’ Erases Disease-Causing Errors

October 25, 2017

(Scientific American) – There are more than 50,000 known human genetic maladies that have, in most cases, few good treatments and no cure. Now researchers at the Broad Institute of Harvard and MIT have developed a new tool that would theoretically make it possible to correct the genetic errors behind about 15,000 of these illnesses—including sickle-cell disease, cystic fibrosis and several forms of congenital deafness and blindness. Standard gene-editing tools, such as the well-known CRISPR–Cas9 system, function like scissors; they can cut an offending gene from a strand of DNA. This could be useful against diseases such as Huntington’s, which is caused by duplications of genetic material. The new tool, called ABE (adenine base editors), is more like an editing pencil, according to lead researcher David Liu.

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