Determination of the retinal functions of VPS13B using a murine model and assessment of exon-skipping as a therapeutic strategy for Cohen Syndrome in patient-derived cells

MDBRCohen
Written By Million Dollar Bike Ride

Awardee: Laurence Faivre

Institution: CHU Dijon Bourgogne

Award Amount: $66,560

Funding Period: February 1, 2021 - January 31, 2022

Million Dollar Bike Ride
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The “molecular tweezer” CLR01 as a new potent drug candidate to slow CNS pathology progression in MPS-I