New Breakthrough Allows Gene Therapy Delivery to Brain Safely

A team of researchers at UT Southwestern Medical Center has made a groundbreaking discovery that could revolutionize the treatment of brain diseases. In a new study, they found that using nitrous oxide, a commonly used analgesic gas, can temporarily open the blood-brain barrier (BBB) to allow safe delivery of gene therapy to the brain.

The BBB is a highly selective border of semipermeable cells that protects the brain from toxins and infections. However, it 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. To overcome this challenge, researchers have been working for decades to develop solutions that can temporarily open the BBB.

In the new study, researchers combined focused ultrasound with nitrous oxide to achieve this goal. The approach significantly reduced the concentrations of microbubbles and FUS pressure needed to open the BBB, posing less risk to brain tissue.

The results showed that nitrous oxide required up to 1,000 times lower concentrations of microbubbles and significantly lower FUS pressure compared to medical air. This new approach was proven effective in delivering a gene that produces a glowing green protein, resulting in greater uptake of the gene than when breathing air.

The researchers’ next step will be to safely test this approach in clinical trials. This breakthrough has the potential to advance care for diseases of the brain that can be treated by targeted therapeutic delivery.

Source: https://www.utsouthwestern.edu/newsroom/articles/year-2025/may-analgesic-gas-blood-brain-barrier.html