High-throughput cellular analytics to identify central nervous system targets for gene therapy

Gene therapy, a technique to manipulate genes in cells of afflicted organs, has recently led to a plethora of transformative treatments. This approach has been successful for simple or self-renewing tissues (e.g. immune system). However, these therapies fall far short for highly complex and non-regenerating organs such as the brain, which contains more than 2,000 distinct cell types regulating essential functions like eating, breathing, and pain. In my talk, I will discuss how high-throughput single cell gene expression profiling data can bridge challenges in designing targeted gene therapies for the central nervous system. I will cover:
*How to use cell profiling data to identify potential targets for gene therapy;
*How to map functions to cell types using stimulus mapping with cellular profiling technology;
*And finally how to find suitable genomic targets for the design of cell type targeted therapies based on these data.
I will bring examples addressing modulation of central pain pathways. However, the introduced workflows are relevant for a wide variety of disease conditions and, in light of the emerging databases characterizing cellular complexity in the human and mouse brains, become relevant for many other disease states.