Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease
Nurselin Ateş,
Secil Akyildiz Demir,
Volkan Seyrantepe
Abstract:Introduction. The Tay-Sachs disease is a progressive neurodegenerative disorder that is caused by a genetic mutation in the HEXA gene coding the lysosomal α-subunit of β-hexosaminidase A. Currently, there is no effective treatment for Tay-Sachs. Induction of exocytosis as a potential treatment approach is suggested to restore lysosomal enlargement in several lysosomal storage diseases. Here, we aimed to test the therapeutic potential of two small molecules, δ-tocopherol and hydroxypropyl-β-cyclodextrin, in fib… Show more
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