Browsing by Subject "AAV Adeno-Associated Virus"
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Long Term Correction of GM2 Gangliosidoses in Sandhoff Mice Through Intravenous Neonatal Gene Therapy, Using a Recombinant Adeno-Associated Viral Vector Expressing a New Hexosaminidase Variant
(2015-12-14)GM2 gangliosidoses are a group of neurodegenerative disorders, characterized by the malfunctioning β-Hexosaminidase A (HexA) enzyme, for which there is no current treatment. HexA is composed of two similar, non-identical ...