NIH Reporter #5R01NS138537-03

Interactions Between Diverse Brain Cell Types Drive Aicardi-Goutieres Neuropathology

This NIH grant funds research to identify the cellular drivers of neuropathology in Aicardi Goutières syndrome using rodent models and immune targeting, aiming to develop targeted therapies for this severe brain disease.

Description

Aicardi Goutières syndrome (AGS) is a severe autoinflammatory disease that predominantly affects the brain, leading to severe cognitive and physical disabilities. This study aims to identify the “driver” cell in either the peripheral or CNS immune system or at brain vascular interfaces that converts genetic mutations into progressive, IFN-mediated neuronal and oligodendrocyte injury. Using rodent models with AGS patient mutations, viral targeting approaches, and immune chimeric models, the project will test which cell(s) promote neuropathology and determine which compartment requires rescue for treatment.

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