Science Spotlight: Brain endothelial cell AAVs, prenatal ASO for SMA

A team from University of California Irvine developed adeno-associated viral (AAV) vectors targeting brain endothelial cells, which could become vectors for delivering transgenes to treat neurological diseases involving the blood-brain barrier. Endothelial cells line the blood vessels in the brain and therefore play a central role in blood-brain barrier function. The authors cite Alzheimer’s disease and traumatic brain injury as potential therapeutic applications for the vectors.

Using genomic and epigenomic analyses, the authors of the study in Neuron identified an enhancer with regions of open chromatin that likely function as regulatory elements in brain endothelial cells. To restrict transgene expression to brain endothelial cells, the group incorporated the enhancer upstream of a minimal promoter in the AAV vector. It had little off-target expression in neurons or other non-vascular cells of the brain. ...