NIH R01 · 2025
Huntington’s disease (HD) is a fatal, dominantly inherited neurodegenerative disorder that primarily affects neurons in the striatum and cortex. Studies have identified striatal developmental impairments in HD and transcriptional pathways that are amenable to disease-modifying interventions. Our central hypothesis is that HD MSNs manifest developmental alterations that cause their dysfunction, dedifferentiation and potentially dysregulation with inappropriate neuronal markers for MSNs. We further hypothesize that these HD phenotypes can be corrected by modulating MSN developmental transcription factors or via drugs that correct transcriptional dysregulation. Specifically, the failure of HD…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.