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

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FUNCTIONALITY OF A BICISTRONIC CONSTRUCTION CONTAINING HEXA AND HEXB GENES ENCODING β-HEXOSAMINIDASE A FOR CELL-MEDIATED THERAPY OF GM2 GANGLIOSIDOSES

Форма представления

Статьи в зарубежных журналах и сборниках

Год публикации

2022

Язык

английский

Авторы, сотрудники КФУ

Аймалетдинов Александр Маазович, автор

Ризванов Альберт Анатольевич, автор

Соловьева Валерия Владимировна, автор

Чулпанова Дарья Сергеевна, автор

Авторы, аспиранты КФУ

Шаймарданова Алиса Алмазовна, автор

Библиографическое описание на языке оригинала

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 gene therapy for Sandhoff disease and Sandhoff disease using a bicistronic lentiviral vector encoding cDNA of HexA α- and β-subunit genes separated by the nucleotide sequence of a P2A peptide (HEXA-HEXB). The functionality of the bicistronic construct containing the HEXA-HEXB genetic cassette was analyzed in a culture of HEK293T cells and human umbilical cord blood mononuclear cells (hUCBMCs). Our results showed that the enzymatic activity of HexA in the conditioned medium harvested from genetically modified HEK293T-HEXA-HEXB and hUCBMCs-HEXA-HEXB was increased by 23 and 8 times, respectively, compared with the conditioned medium of native cells. Western blot analysis showed that hUCBMCs-HEXA

Ключевые слова

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

Название журнала

Neural Regeneration Research

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https://repository.kpfu.ru/?p_id=268794

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Полная запись метаданных

Поле DC	Значение	Язык
dc.contributor.author	Аймалетдинов Александр Маазович	ru_RU
dc.contributor.author	Ризванов Альберт Анатольевич	ru_RU
dc.contributor.author	Соловьева Валерия Владимировна	ru_RU
dc.contributor.author	Чулпанова Дарья Сергеевна	ru_RU
dc.contributor.author	Шаймарданова Алиса Алмазовна	ru_RU
dc.date.accessioned	2022-01-01T00:00:00Z	ru_RU
dc.date.available	2022-01-01T00:00:00Z	ru_RU
dc.date.issued	2022	ru_RU
dc.identifier.citation	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 gene therapy for Sandhoff disease and Sandhoff disease using a bicistronic lentiviral vector encoding cDNA of HexA α- and β-subunit genes separated by the nucleotide sequence of a P2A peptide (HEXA-HEXB). The functionality of the bicistronic construct containing the HEXA-HEXB genetic cassette was analyzed in a culture of HEK293T cells and human umbilical cord blood mononuclear cells (hUCBMCs). Our results showed that the enzymatic activity of HexA in the conditioned medium harvested from genetically modified HEK293T-HEXA-HEXB and hUCBMCs-HEXA-HEXB was increased by 23 and 8 times, respectively, compared with the conditioned medium of native cells. Western blot analysis showed that hUCBMCs-HEXA	ru_RU
dc.identifier.uri	https://repository.kpfu.ru/?p_id=268794	ru_RU
dc.description.abstract	Neural Regeneration Research	ru_RU
dc.language.iso	ru	ru_RU
dc.subject		ru_RU
dc.title	Functionality of a bicistronic construction containing HEXA and HEXB genes encoding β-hexosaminidase A for cell-mediated therapy of GM2 gangliosidoses	ru_RU
dc.type	Статьи в зарубежных журналах и сборниках	ru_RU