Gene editing... quo vadis?

Abstract

As discussed in the last editorial, modern gene editing is quite precise but it is not perfect. The procedure can be hit and miss, reaching some cells but not others. Even when Crispr gets where it is needed, the edits can differ from cell to cell, for example mending two copies of a mutated gene in one cell, but only one copy in another. For some genetic diseases this may not matter, but it may if a single mutated gene causes the disorder. Another common problem happens when edits are made at the wrong place in the genome. There can be hundreds of these “offtarget” edits that can be dangerous if they disrupt healthy genes or crucial regulatory DNA.