Here’s a bold statement: the future of gene editing for brain disorders might not be as straightforward as we thought. But here's where it gets controversial: while CRISPR-Cas9 has been hailed as a revolutionary tool for treating genetic diseases, its application in neurons and other nondividing cells is far more complex than anyone anticipated. And this is the part most people miss—the very cells we’re trying to fix in brain disorders behave fundamentally differently when it comes to DNA repair.
In a groundbreaking study published in Nature Communications (https://www.nature.com/articles/s41467-025-66058-3), researchers from Gladstone Institutes, the Innovative Genomics Institute, and UC Berkeley uncovered why gene editing in neurons has been so challenging. Led by Gladstone Senior Investigator Bruce Conklin, MD, the team found that neurons and other nondividing cells respond uniquely to CRISPR-Cas9 compared to dividing cells. The key takeaway? The rules of the genome editing playbook need a rewrite for nondividing cells.
