CORDIS Project
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This project focuses on developing a gene therapy strategy for GM1-gangliosidosis, a severe neurodegenerative disorder. It aims to deliver therapeutic genes to the central nervous system to alleviate symptoms and improve outcomes in affected individuals.
GM-gangliosidosis (OMIM #230500) is a rare, autosomal recessive, neurodegenerative Lysosomal Storage Disorder.
It is caused by mutations in the GLB1 gene, encoding the lysosomal hydrolase β-galactosidase.
Infantile GM1-gangliosidosis is characterized by neurodevelopmental delay, hypotonia, dysphagia, seizures and death by 3 years of life.
Due to the rapid progression and severe nature of this disease, which involves storage of undegraded metabolites and secondary mechanisms of cell damage, corre…
UNIVERSITA DEGLI STUDI DI PADOVA
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