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

Awardee: Jan Wijnholds

Institution: Leiden University Medical

Award Amount: $40,177

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

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