As gene editing technologies using CRISPR advance, scientists increasingly need ways to
Doctors from the Broad Institute and Brigham and Women's Hospital said in a new study that they have found molecules that can safely and quickly block the process of gene editing using CRISPR.
According to the senior author of the new studyAmita Chodie, in similar works, other scientists are looking for CRISPR inhibitors among large proteins, while this group of doctors is focused on exploring the possibilities of using synthetic low-molecular objects.
As part of the study, scientists have collected a platformfor screening, which can accurately measure the activity of the CRISPR enzyme inside the cell. The neural network analyzed more than 15 thousand potential biological compounds and their reactions to CRISPR in order to identify the most effective ones for inhibition.
One compound, BRD0539, was able toinhibit the Cas9 enzyme and unbind it from DNA. Its activity was highly dose-dependent, which will make it possible in the future to control the degree of inhibition almost directly. In addition, BRD0539 was found to be stable in plasma and easily removed from blood.
Scientists note that while molecules cannot be used in clinical trials, because genetics have only begun to investigate the use of BRD0539 in the inhibition process.