Researchers at Rice University in the United States have unveiled a non-invasive brain gene‑therapy approach that leverages focused ultrasound to ferry genetic material to targeted brain regions. In this new method, genes are delivered through the skull via ultrasound rather than by traditional injections, a technique that early tests suggest may reach wider areas of the brain more efficiently and with less tissue disruption. EurekAlert has reported on these findings, highlighting the potential for safer and more effective gene transfer across complex neural networks.
Gene therapy encompasses a range of genetic engineering and medical tools aimed at correcting or compensating for DNA defects in human somatic cells. The goal is to introduce a functional gene to compensate for faulty instructions within cells. In the brain, many disorders affect large swaths of tissue, making uniform gene delivery challenging. Conventional needle injections typically spread just a few millimeters from the injection site, meaning that thousands of separate injections could be required to cover a meaningful portion of neural tissue. That reality raises concerns about procedure-associated risks and practicality. The Rice team’s ultrasound-assisted approach promises broader distribution while potentially minimizing invasiveness and harm, making it a promising avenue for treating widespread neurodegenerative or neurological conditions.
Shirin Nurain, the study’s lead author and a doctoral student in the lab of bioengineer Jerzy Sablowski, described the mechanism as opening nanoscale pores in the brain’s endothelium—the thin layer of cells lining the interior surface of blood vessels throughout the brain. This endothelial layer forms a selective barrier that governs what can pass from the bloodstream into brain tissue. By transiently widening these barriers with focused ultrasound, researchers aim to create a temporary conduit that allows therapeutic genes to reach neural cells more evenly and efficiently than injections alone.
The work has progressed to in vivo testing in mice, where researchers observed that expanding the ultrasound treatment to additional brain regions could boost gene uptake without compromising safety. Importantly, the team reported no evidence of neuron loss when multiple regions of the rodents’ brains were stimulated in a single session, suggesting a favorable safety profile at the tested parameters. While these results are encouraging, scientists acknowledge the next steps involve refining the technique, ensuring precise targeting, and evaluating long-term outcomes to determine whether this approach can be translated into human therapies for neurological diseases.