Adeno-Associated Virus Vector Mediated Gene Delivery to Neurons and Glial Cells in Damaged Spinal Cord

Abstract Recombinant adeno-associated virus (AAV) vectors have emerged as a potentially useful gene delivery approach for neurons in a number of neurodegenerative conditions, including spinal cord injury (SCI). Since the role of glia plays an important role in the progression of “secondary damage,” we searched for the optimal vectors for gene transfer to both neurons and glial cells following contusion SCI in adult rats. Comparing transduction efficacy by various AAV serotypes, we demonstrate that AAV-rh10 transduced significantly higher number of macrophages/microglia and oligodendrocytes in damaged spinal cord. Efficacy to transduce neurons was comparable to other AAV serotypes. Thus AAV-rh10 carries promising potential as a gene therapy vector, particularly if both the neuronal and glial cell populations in damaged spinal cord are targeted. Recent studies revealed that AAV-rh10 is a promising vector for therapeutic transgene delivery to damaged spinal cord: AAV-rh10 vector mediated delivery of neurotrophin NT-3 facilitated recovery of synaptic transmission and function following SCI.