Therapeutic developments for neurodegenerative GM1 gangliosidosis
Article in Frontiers in Neuroscience (April 2024)
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Silencing of B4Galnt1 Gene Prevents GM2 Accumulation in Tay-Sachs Cells
Article in Advances in Cell and Gene Therapy (March 2024)
Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders
Article in International Journal of Molecular Sciences (January 2024)