CSF dynamics in mucopolysaccharidosis type I

Awardee: Igor Nestrasil

Institution: University of Minnesota

Grant Amount: $60,655.00

Funding Period: February 1, 2024 - January 31, 2025


Summary:

This project aims to measure the motion of the cerebrospinal fluid (CSF) through the brain with magnetic resonance imaging (MRI) and associate these measures with disease severity in mucopolysaccharidosis type I (MPS I). The findings may determine whether CSF motion is impaired in MPS I with implications for the disease severity or the drug distribution administered to the intrathecal space (space surrounding the spinal cord) or subarachnoid space (space around the brain).

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SKIP-AT: a comprehensive, systematic platform approach for targeted exon skipping of ATM

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To Study the potential for Collagen Mimetic Peptides to reduce progressive damage to the Bruch's-RPE-Photoreceptor Complex in Choroideremia.