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Assessing Juvenile Batten Disease on Brain Development in In Vitro 3D Organoid Models


Neuronal ceroid lipofuscinosis (NCL) is a complex, rare, and fatal disease belonging to a larger group of lysosomal storage disorders that presents with nervous system symptoms. Batten disease – or CLN3 disease – is another name given commonly to Juvenile NCL (JNCL). Dr. Gomez-Giro and her team focused on understanding the neurodevelopmental process of JNCL utilizing healthy hiPSCs introduced with a disease-causing c.1054C→T pathologic variant into the CLN3 gene. Continue reading to find out more about Dr. Gomez-Giro's research.