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Increasing β-hexosaminidase A activity using genetically modified mesenchymal stem cells

GM2 gangliosidoses are a group of autosomal-recessive lysosomal storage disorders. These diseases result from a deficiency of lysosomal enzyme β-hexosaminidase A (HexA), which is responsible for GM2 ganglioside degradation. HexA deficiency causes the accumulation of GM2-gangliosides mainly in the ne...

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Detalles Bibliográficos
Autores principales:	Shaimardanova, Alisa A., Chulpanova, Daria S., Solovyeva, Valeriya V., Issa, Shaza S., Mullagulova, Aysilu I., Titova, Angelina A., Mukhamedshina, Yana O., Timofeeva, Anna V., Aimaletdinov, Alexander M., Nigmetzyanov, Islam R., Rizvanov, Albert A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wolters Kluwer - Medknow 2023
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10479847/ https://www.ncbi.nlm.nih.gov/pubmed/37488869 http://dx.doi.org/10.4103/1673-5374.375328

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