Utilizing the PS Gene Editing System to Encode a Blood‐Brain Barrier‐Penetrating IDUA as a Treatment for Murine Hurler Syndrome

MPS2022MPSI
Written By Million Dollar Bike Ride

Awardee: Michael Przybilla

Institution: University of Minnesota

Award Amount: $165,000

Funding Period: February 1, 2023 - January 31, 2024

Million Dollar Bike Ride
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Biochemical investigation of NUBPL disease mechanisms and therapy candidates
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The new lysosomal activator GHF201 (144DG11) as a potential treatment for mucopolysaccharidosis I”