Scientists at Virginia Tech’s Fralin Biomedical Research Institute at VTC have discovered how an experimental therapy can help brain cells overcome the effects of a disease-causing genetic deletion. Instead of repairing the deletion and its immediate consequences, the therapy redirects brain development by helping at-risk neurons grow and connect more normally.

The findings, published in Disease Models & Mechanisms, suggest that some genetic brain disorders may be treated by targeting the cellular mechanisms disrupted by a genetic change rather than repairing the genetic change itself.