Presentation description

Vanishing White Matter (VWM) disease is a rare leukodystrophy affecting about 1 in every 80,000 births and is characterized by spasticity, speech issues, cognitive deficits, and more. As there is no cure, our research aims to further our understanding of VWM and investigate astrocyte-specific promoters for AAV9 gene therapy in a mouse model. The development of a viable treatment is paramount in offering hope and therapeutic intervention to individuals grappling with this debilitating disease.