Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

GM2 gangliosidosis is a family of three genetic neurodegenerative disorders caused by the accumulation of GM2 ganglioside (GM2) in neuronal tissue. Two of these are due to the deficiency of the heterodimeric (α-β), "A" isoenzyme of lysosomal β-hexosaminidase (HexA). Mutations in the α-subu...

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Veröffentlicht in:	Human gene therapy 2016-07, Vol.27 (7), p.509-521
Hauptverfasser:	Karumuthil-Melethil, Subha, Nagabhushan Kalburgi, Sahana, Thompson, Patrick, Tropak, Michael, Kaytor, Michael D, Keimel, John G, Mark, Brian L, Mahuran, Don, Walia, Jagdeep S, Gray, Steven J
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Animals Animals, Newborn Brain Central nervous system Dependovirus - genetics Design Disease Disease Models, Animal Female G(M2) Ganglioside - metabolism Ganglioside GM2 Gangliosidosis Gene transfer Genetic Therapy Genetic Vectors - administration & dosage Genomes Hexosaminidases - genetics Liver Liver - metabolism Medical treatment Mice Mice, Inbred C57BL Mutation Mutation - genetics Neonates Nervous system Neurodegenerative diseases Packaging Proteins Spinal cord Substrates Tay-Sachs disease Tay-Sachs Disease - genetics Tay-Sachs Disease - therapy Viruses
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