Characterisation of CRB1 patient human iPSC-derived retinal organoids to set-up potency assays for gene therapy

MDBRCRB1

Feb 1

Written By Million Dollar Bike Ride

Awardee: Jan Wijnholds

Institution: Leiden University Medical

Award Amount: $40,177

MDBRCRB12019

Million Dollar Bike Ride

A study of the correlation between gait abnormalities, activity monitoring parameters, CMTPeds and a biomarker in children with Charcot-Marie-Tooth disease

Pre-malignant Clonal Evolution in Telomere Biology Disorders (TBD)