Charting WDR45-dependent genes for neurodegeneration at a single-cell resolution

Awardee: Young Ah Seo

Institution: Regents of the University of Michigan

Grant Amount: $75,815.00

Funding Period: February 1, 2025 - January 31, 2026

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Summary:

BPAN (β-propeller protein-associated neurodegeneration) is a rare neurodegenerative disorder caused by mutations in the WDR45 gene, often leading to progressive brain damage and abnormal iron buildup in the brain. Unfortunately, no cure exists for BPAN, and current treatments only manage symptoms. Our research focuses on uncovering the underlying causes of neurodegeneration in BPAN. Using a mouse model where the WDR45 gene has been deleted, we will employ single-cell RNA sequencing (scRNA-seq) to analyze changes in gene expression at the individual cell level. This will help us identify the specific brain cells and molecular pathways most affected by WDR45 loss. By mapping these pathways, we aim to pinpoint new targets for potential therapies that could prevent or slow down the progression of BPAN. This project could pave the way for developing treatments that address the root causes of BPAN, benefiting patients with this rare condition.