New Gene Delivery Vehicle (Engineered AAV) Shows Promise for Human Brain Gene Therapy

Scientists have engineered an adeno-associated virus (AAV) that efficiently crosses the blood-brain barrier in human cell models and delivers genes throughout the brain in humanized mice

Graphic image of adeno-associated virus (AAV)
In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein. Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients. The work was published in Science on May 16, 2024. The article is Titled “An AAV Capsid Reprogrammed to Bind Human Transferrin Receptor Mediates Brain-Wide Gene Delivery.”
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