Cargando…
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...
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: | |
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 |
Ejemplares similares
-
Increasing β-hexosaminidase A activity using genetically modified mesenchymal stem cells
por: Shaimardanova, Alisa A., et al.
Publicado: (2023) -
Identification of a novel HEXB Mutation in an Iranian Family with suspected patient to GM2‐gangliosidoses
por: Mansouri‐Movahed, Fatemeh, et al.
Publicado: (2020) -
Serum Cytokine Profile, Beta-Hexosaminidase A Enzymatic Activity and GM(2) Ganglioside Levels in the Plasma of a Tay-Sachs Disease Patient after Cord Blood Cell Transplantation and Curcumin Administration: A Case Report
por: Shaimardanova, Alisa A., et al.
Publicado: (2021) -
Current Strategies for the Gene Therapy of Autosomal Recessive Congenital Ichthyosis and Other Types of Inherited Ichthyosis
por: Chulpanova, Daria S., et al.
Publicado: (2022) -
Combined replacement effects of human modified β-hexosaminidase B and GM2 activator protein on GM2 gangliosidoses fibroblasts
por: Kitakaze, Keisuke, et al.
Publicado: (2016)