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Functionality of a bicistronic construction containing HEXA and HEXB genes encoding β-hexosaminidase A for cell-mediated therapy of GM2 gangliosidoses

Tay-Sachs disease and Sandhoff disease are severe hereditary neurodegenerative disorders caused by a deficiency of β-hexosaminidase A (HexA) enzyme, which results in the accumulation of GM2 gangliosides in the nervous system cells. In this work, we analyzed the efficacy and safety of cell-mediated g...

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Detalles Bibliográficos
Autores principales:	Shaimardanova, Alisa A., Chulpanova, Daria S., Solovyeva, Valeriya V., Aimaletdinov, Aleksandr M., Rizvanov, Albert A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wolters Kluwer - Medknow 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8451576/ https://www.ncbi.nlm.nih.gov/pubmed/34100447 http://dx.doi.org/10.4103/1673-5374.314310

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