New Platform for Treating Neurodegeneration and Brain Disorders Developed by University of Pittsburgh Neurobiologist

Thursday - April 4 2024, 19:23 UTC - 10 months ago

University of Pittsburgh neurobiologist receives grant to develop platform for manipulating glia cells in the brain and potentially treat neurodegeneration and brain disorders. The platform uses modified RNAs to deliver genes, bypassing the need for viruses and reducing the risk of immune responses. Initial testing will be conducted in mouse brains and could have a significant impact on treating conditions such as Alzheimer's disease.

Or Shemesh, Ph.D., an assistant professor of neurobiology at the University of Pittsburgh, has received a three-year, $400,000 Trailblazer Award from the National Institute of Biomedical Imaging and Bioengineering (NIBIB), a division of the National Institutes of Health (NIH). This grant will support the development of a new platform aimed at potentially treating neurodegeneration and brain disorders.

The platform focuses on manipulating glia cells, a class of cells that support and protect neurons in the brain. Shemesh explains, "Glia cells are pivotal in the development of brain diseases, so altering their activity could lead to new treatments." While scientists have had success using virus-based vectors to deliver genes to neurons, this approach has not been as effective in glia cells, which include astrocytes, oligodendrocytes, microglia, and other cell types.

To address this, Shemesh and his team are using bioengineering techniques to create modified RNAs, or modRNAs, that can be directly translated into proteins in the cell. This bypasses the risk of triggering an immune response, which can occur when using a virus to deliver genes.

In order to demonstrate the efficacy of their platform, the team will use their glia RNA-vector technology to manipulate the activity of disease-relevant genes in astrocytes and microglia in the brains of mice. Shemesh states, "We are seeing more research, including from Pitt, that suggests astrocytes and other glia play a crucial role in the development of Alzheimer's disease. So our new platform could one day provide a unique strategy to treat dementia." .

This groundbreaking research was made possible by the support of the NIBIB. As the platform continues to be developed and tested, it could potentially provide a novel approach to treating neurodegenerative diseases and other brain disorders.