Developing viral vectors for applications in neuroscience
Gene delivery to nervous tissues is not only important for neuroscience research but also a potential means for the treatment of neurological disorders. However, most cell types relevant to nervous system disorders are difficult to transfect using non-viral transfection and traditional viral transduction methods. Genetic manipulation of brain cells in vivo has been even more challenging. Lentiviral vectors are able to transduce non-dividing cells, including neurons, making them attractive vehicles for gene delivery to neural cells. Several adeno-associated virus (AAV) serotypes have also been shown to successfully transduce cultured neurons and to mediate robust transgene expression in the brain.
We are developing new vectors for neurological applications. The goal of the Viral Vectors Core is to assist Washington University neuroscience researchers in the design and production of various kinds of vectors. Currently, we are focused on lentiviral and AAV vectors for use in cell culture and in vivo pre-clinical experiments designed to understand the causes and treatments for nervous system disorders. Among the projects we now undertaking are developing tissue-specific expression vectors and incorporating viral vectors with RNAi technology. We will also aim to promote translation of preclinical research findings into clinical applications.