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

MDBRCDKL5
Written By Million Dollar Bike Ride

Awardee: Maurizio Giustetto

Institution: Univ. of Torino - Dept. of Neuroscience

Grant Amount: $75,000

Funding Period: February 1, 2022 - January 31, 2023

Summary:

The identification of objectively measurable parameters is urgently needed to speed up the diagnosis of CDKL5 deficiency disorder (CDD) and to evaluate the outcomes of both pre-clinical and clinical trials. Although exosomes, nanovescicles seceted by all cell types, can be exploited as unbiased, quantitative and non-invasive biomarker for clinical diagnosis, so far, no information is available on the molecular profile of exosomes in CDD. The goal of our proposal is to fill this gap and by establishing and validating a novel molecular, reliable biomarker for CDD patients.

Million Dollar Bike Ride
Previous

Discovery of new therapies for hypertrophic cardiomyopathy in RASopathies using an innovative hiPSC-based high-throughput drug screening platform
Next

ASO therapy in CDKL5 deficiency disorder