US scientists based at the University of California School of Medicine, Los Angeles (UCLA), have developed a new, non-invasive technique for delivering genes to the brain. It opens the way for gene therapies for Parkinson's and Alzheimer's disease, as well as genetic conditions such as Gaucher's and Tay Sach's disease. The research is published in the latest issue of the Proceedings of the National Academy of Sciences.
Gene therapy aims to treat diseases by delivering genes to the affected body tissues, where they make protein that alleviate the disease symptoms. But delivering genes to the brain has proved problematic because of the blood-brain barrier, which protects the brain from potentially dangerous substances. Previous attempts to get genes into the brain have included drilling holes in the patient's skull, and injecting irritants into the artery that supplies blood to the brain.
The UCLA team, led by Dr William Partridge and Ningya Shi, packaged the genes into tiny fatty spheres, called liposomes. The liposomes were attached to proteins recognised by cells that make up the blood-brain barrier. In this way, the scientists managed to 'smuggle' the gene packages into rat brains.
Partridge said his team had developed a similar delivery system that would soon be ready to test in humans.
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Researchers sneak gene therapy into brain