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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Egile Nagusiak:	Alisa A Shaimardanova, Daria S Chulpanova, Valeriya V Solovyeva, Aleksandr M Aimaletdinov, Albert A Rizvanov
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	Wolters Kluwer Medknow Publications 2022-01-01
Saila:	Neural Regeneration Research
Gaiak:	bicistronic vector; cell-mediated gene therapy; gm2 gangliosidosis; p2a peptide; sandhoff disease; tay-sachs disease; umbilical cord blood mononuclear cells; β-hexosaminidase
Sarrera elektronikoa:	http://www.nrronline.org/article.asp?issn=1673-5374;year=2022;volume=17;issue=1;spage=122;epage=129;aulast=Shaimardanova
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