Blood-brain barrier bypassed

Scientists use gene therapy and low-density lipoprotein receptors to deliver drugs to the mouse brain

Chandra Shekhar
Using a combination of gene therapy and receptor targeting, scientists have successfully enabled a protein to cross the blood-brain barrier into the central nervous system, a crucial step in delivering drugs to diseased brains, according to a study in this week's Proceedings of the National Academy of Sciences. "It's a very nice demonstration of how to get proteins into the brain," David Begley of the Blood-Brain Barrier Group at King's College in London, who was not involved in the study, told The Scientist. During the study, the researchers delivered the enzyme glucocerebrosidase to the brains of adult mice. In humans, a deficiency of this enzyme causes Gaucher's disease, a genetic disorder that is often neurodegenerative and fatal.Made of tightly packed endothelial cells that line capillaries in the brain, the blood-brain barrier permits only a few essential chemicals to enter the brain. The barrier makes neural disorders such as...

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