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

Neural Regen Res .. 2024-01; 
Alisa A Shaimardanova , Daria S Chulpanova , Valeriya V Solovyeva 1, Shaza S Issa , Aysilu I Mullagulova , Angelina A Titova , Yana O Mukhamedshina , Anna V Timofeeva , Alexander M Aimaletdinov , Islam R Nigmetzyanov , Albert A Rizvanov
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Gene Synthesis The OptimumGene algorithm (GenScript, Piscataway, NJ, USA) was used for codon optimization of HEXA and HEXB genes. De novo synthesis of cDNA nucleotide sequences for human HEXA and HEXB genes was carried out by GenScript (Piscataway, NJ, USA). Cloning HEXA, HEXB, and Katushka2S (encoding far-red fluorescent protein) genes into the vector plasmid pAAVMCS (Agilent Technologies, Santa Clara, CA, USA), was carried out by GenScript (Piscataway, NJ, USA) Get A Quote

Abstract

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 nervous system cells, leading to severe progressive neurodegeneration and neuroinflammation. To date, there is no treatment for these diseases. Cell-mediated gene therapy is considered a promising treatment for GM2 gangliosidoses. This study aimed to evaluate the ability of genetically modified mesenchymal stem cells (MSCs-HEXA-HEXB) to restore HexA deficiency in Tay-Sachs disease patient cells, as well as to analyze ... More

Keywords

GM2 gangliosidosis; Sandhoff disease; Tay-Sachs disease; adeno-associated viral vectors; cell therapy; cell-mediated gene therapy; gene therapy; β-hexosaminidase.