AAV-BR1 Does Not Target Endothelial Cells in Sprague Dawley Rats Unlike in Mice.

Adeno-associated viruses (AAVs) are a popular tool in gene therapy approaches and have been engineered to specifically target different cells. There is interest in targeting endothelial cells (ECs) of the blood brain barrier and the AAV2 capsid variant BR1 has been found to transduce ECs with high selectivity in various mice models. However, this has not been tested in rat models. Here, we show that there is no EC transduction with systemic injection of the AAV-BR1-CAG-GFP virus in Sprague-Dawley rats (n=3), but instead transduction of brain parenchymal cells with neuronal morphology. These findings emphasize the importance of species-differences in use of AAVs.