Researchers from Rice University and Baylor College of Medicine produced a split gene editing tool that stays inactive when disassembled but activates when the binding molecule is added. This tool has better precision and is easier to control compared to the intact version.
Single base-pair mutations or point mutations can cause thousands of diseases in humans. To help solve this issue, the researchers developed a tool that can accurately edit a single base pair on a Chosen gene. It was designed to target adenine, which is one of the four main building blocks of DNA.
The adenine base editor was separated into two proteins that stay inactive until sirolimus (the binding molecule) is added. This enabled an on & off switch to increase the safety, accuracy, and precision of the tool, as well as prevent off-target edits. Moreover, sirolimus is already being used as an anticancer and immunosuppressive drug. In the future, the tool could have broad therapeutic applications.