Award
NIH Reporter #1R21HD119649-01
Role of Microglia in Vanishing White Matter Disease Leukodystrophy
Recipient
Utah State Higher Education System--University of Utah, Salt Lake City, UT
Award Amount
$424,655.00
Ceiling
$424,655.00
Awarded
August 22, 2025
Identifier
1R21HD119649-01
This award funds research on the role of microglia in Vanishing White Matter Disease, aiming to improve gene therapy outcomes by combining microglia replacement with existing treatments.
Description
Leukoencephalopathy with Vanishing White Matter (VWM) is a rare neurodegenerative leukodystrophy caused by mutations in EIF2B genes, leading to persistent ISR activation. Current gene therapy approaches target astrocytes but show waning efficacy, possibly due to activated microglia contributing to disease progression. The project aims to characterize microglial involvement and test microglia replacement combined with gene therapy to improve outcomes.