The new lysosomal activator GHF201 (144DG11) as a potential treatment for mucopolysaccharidosis I”

MPS2022MPSI

Feb 1

Written By Million Dollar Bike Ride

Awardee: Or Kakhlon

Institution: Research Fund of the Hadassah Medical Organization

Award Amount: $165,000

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

MPSMPSI2022

Million Dollar Bike Ride

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

Choroid plexus disruption in CDD