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Ex vivo gene therapy for GM1-gangliosidosis

Project description

Ex vivo gene therapy for GM1-gangliosidosis

GM1-gangliosidosis is an inherited disorder caused by mutations in the GLB1 gene that encodes the lysosomal hydrolase β-galactosidase. Patients accumulate undegraded metabolites in lysosomes which triggers cell damage, affecting mainly the central nervous system. The EU-funded GT-GM1 project proposes to develop a gene therapy approach for delivering the GLB1 gene alone or in association with a neuroprotective factor. Scientists will employ a mouse model of the disease and lentiviral vectors for the ex vivo delivery of the gene to hematopoietic stem/progenitor cells. Genetically modified cells will be re-infused intravenously and into the brain ventricles of animals for a therapeutic effect.

Coordinator

UNIVERSITA DEGLI STUDI DI PADOVA
Net EU contribution
€ 171 473,28
Address
Via 8 Febbraio 2
35122 Padova
Italy

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Region
Nord-Est Veneto Padova
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00