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.