Functionality of a bicistronic construction containing <i>HEXA</i> and <i>HEXB</i> genes encoding ß-hexosaminidase A for cell-mediated therapy of GM2 gangliosidoses.

Shaimardanova, Alisa A; Chulpanova, Daria S; Solovyeva, Valeriya V; Aimaletdinov, Aleksandr M; Rizvanov, Albert A

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

Shaimardanova, Alisa A; Chulpanova, Daria S; Solovyeva, Valeriya V; Aimaletdinov, Aleksandr M; Rizvanov, Albert A.

Afiliação

Shaimardanova AA; Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan, Russia.
Chulpanova DS; Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan, Russia.
Solovyeva VV; Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan, Russia.
Aimaletdinov AM; Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan, Russia.
Rizvanov AA; Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan, Russia.

Neural Regen Res ; 17(1): 122-129, 2022 Jan.

Article em En | MEDLINE | ID: mdl-34100447

Palavras-chave

GM2 gangliosidosis; P2A peptide; Sandhoff disease; Tay-Sachs disease; bicistronic vector; cell-mediated gene therapy; umbilical cord blood mononuclear cells; ß-hexosaminidase

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Revista: Neural Regen Res Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Federação Russa

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