Tay-Sachs disease, a progressive neurodegenerative disorder caused by mutations in the HEXA gene, lacks effective treatment. This study explores the therapeutic potential of δ-tocopherol and hydroxypropyl-β-cyclodextrin, targeting lysosomal exocytosis in Tay-Sachs models. Results indicate significant lysosomal GM2 reduction, offering hope for future treatments.
Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease (hindawi.com)
