Nitrous oxide, a commonly used analgesic gas, temporarily improved the opening of the blood-brain barrier (BBB) to allow gene therapy delivery in mouse models using focused ultrasound (FUS), UT Southwestern Medical Center researchers report in a new study. Their findings, published in Gene Therapy, could eventually lead to new ways to treat a variety of brain diseases and disorders.

“The approach we explored in this study has the potential to advance care for diseases of the brain that can be treated by targeted therapeutic delivery,” said study leader Bhavya R. Shah, M.D., Associate Professor of Radiology, Neurological Surgery, and in the Advanced Imaging Research Center at UT Southwestern. He’s also an Investigator in the Peter O’Donnell Jr. Brain Institute and a member of the Center for Alzheimer’s and Neurodegenerative Diseases. Deepshikha Bhardwaj, Ph.D., Senior Research Associate at UTSW, was the study’s first author.

The BBB is a highly selective border of semipermeable cells that line tiny blood vessels supplying blood to the brain. It is thought to have developed during evolution to protect the brain from toxins and infections in the blood. However, the BBB also impedes the delivery of drugs that could be used to treat neurologic or neuropsychiatric conditions, such as Alzheimer’s disease, multiple sclerosis, or brain tumors. Consequently, researchers have worked for decades to develop solutions that can temporarily open the BBB to allow treatments to enter.