ASO therapy in CDKL5 deficiency disorder

Awardee: Michela Fagiolini, PhD

Institution: Boston Children's Hospital

Award Amount: $150,000

Funding Period: May 1, 2021 - April 31, 2022

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EXPLOITING NEURONS- AND BRAIN-DERIVED EXOSOMES AS BIOMARKER FOR CDKL5 DEFICIENCY DISORDER

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Comparison of Brain Biodistribution of Two CDKL5 Gene Therapy Vector Leads