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

G(M2) gangliosidosis is a family of three genetic neurodegenerative disorders caused by the accumulation of G(M2) ganglioside (G(M2)) 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-subunit...

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Detalles Bibliográficos
Publicado en:Hum Gene Ther
Main Authors: Karumuthil-Melethil, Subha, Kalburgi, Sahana Nagabhushan, Thompson, Patrick, Tropak, Michael, Kaytor, Michael D., Keimel, John G., Mark, Brian L., Mahuran, Don, Walia, Jagdeep S., Gray, Steven J.
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc. 2016
Assuntos:
Research Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5349231/
https://ncbi.nlm.nih.gov/pubmed/27197548
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.013
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