Science Spotlight: How an AAV from Receptive crosses the BBB

A California Institute of Technology team led by Viviana Gradinaru engineered AAV capsids that cross the blood-brain barrier (BBB) after systemic administration in a humanized mouse model.

The authors of the Cell Reports paper made capsids that target carbonic anhydrase IV (CAIV), a BBB transcytosis receptor expressed on human brain endothelial cells. The capsids, AAV-hCA4-IV68 and AAV-hCA4-IV77, achieved 100-fold increases in brain transduction compared with AAV9 and had broad neuronal and astrocytic coverage, as shown using a fluorescent reporter gene delivered systemically to mice expressing human CAIV...