Volume 32 Issue 3, May 2025

Cover Credit: AAV-mediated CAS9 delivery to the hippocampus is drastically improved by opening the blood-brain barrier (BBB). Genome editing has an assured place in the future of therapies for diseases of the brain. One major challenge is how to deliver the large editing enzymes such as Cas9. In this issue of Gene Therapy, evidence is presented for dramatic enhancement of viral-mediated delivery through brief and transient opening of the BBB using focused ultrasound. The image shows RNAScope results of Cas9 expression following intrathecal AAV-mediated delivery and opening the BBB of the right but not left hippocampal region.