Adeno-associated viral vectors (AAVs) are widely used for gene transfer to the nervous system. This study compares four promoters (sCAG, hCMV, mPGK, hSYN) packaged in AAV1 for corticospinal tract transduction in rats and mice. hCMV and sCAG resulted in broad expression, while mPGK and hSYN showed stronger, more targeted expression. mPGK drove expression in neurons and oligodendrocytes, whereas hSYN yielded neuron-specific expression, including in layer V corticospinal neurons. This optimized transduction improves gene delivery to the brain and spinal cord, benefiting spinal cord injury research.

Bart Nieuwenhuis, Barbara Haenzi, Sam Hilton, Alejandro Carnicer-Lombarte, Barbara Hobo, Joost Verhaagen, James W. Fawcett