An Adeno-Associated Virus with Enhanced Ability to Cross the Brain-Blood Barrier
July 30, 2024 | Terry Sharrer
“In a new study published in Science, the team showed that their AAV, when injected into the bloodstream in mice expressing a humanized transferrin receptor, crossed into the brain at much higher levels than the AAV that is used in an FDA-approved gene therapy for the central nervous system, AAV9. It also reached a large fraction of important types of brain cells, including neurons and astrocytes. The researchers then showed that their AAV could deliver copies of the GBA1 gene, which has been linked to Gaucher’s disease, Lewy body dementia, and Parkinson’s disease, to a large fraction of cells throughout the brain.” MORE
Image Credit: Deverman Lab, Broad Institute