Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 1 of 1 for “"Gene Theray"”.
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Preclinical Assessment of Intravenous Gene Therapy for GM2 Gangliosidosis
GM2 Gangliosidoses are a group of severe neurodegenerative lysosomal storage disorders characterized by the inability to catabolize GM2 Gangliosides, leading to a neurotoxic accumulation of the GM2 lipids within the central nervous system. The Hexosaminidase A (HexA) enzyme is a heterodimeric …