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J Appl Microbiol ; 123(2): 414-428, 2017 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-28609559

RESUMO

AIMS: Adeno-associated virus type 2 (AAV) is a nonpathogenic parvovirus that is a promising tool for gene therapy. We aimed to construct plasmids for optimal expression and assembly of capsid proteins and evaluate adenovirus (Ad) protein effect on AAV single-stranded DNA (ssDNA) formation in Saccharomyces cerevisiae. METHODS AND RESULTS: Yeast expression plasmids have been developed in which the transcription of AAV capsid proteins (VP1,2,3) is driven by the constitutive ADH1 promoter or galactose-inducible promoters. Optimal VP1,2,3 expression was obtained from GAL1/10 bidirectional promoter. Moreover, we demonstrated that AAP is expressed in yeast and virus-like particles (VLPs) assembled inside the cell. Finally, the expression of two Ad proteins, E4orf6 and E1b55k, had no effect on AAV ssDNA formation. CONCLUSIONS: This study confirms that yeast is able to form AAV VLPs; however, capsid assembly and ssDNA formation are less efficient in yeast than in human cells. Moreover, the expression of Ad proteins did not affect AAV ssDNA formation. SIGNIFICANCE AND IMPACT OF THE STUDY: New manufacturing strategies for AAV-based gene therapy vectors (rAAV) are needed to reduce costs and time of production. Our study explores the feasibility of yeast as alternative system for rAAV production.


Assuntos
Proteínas do Capsídeo/genética , DNA de Cadeia Simples/genética , Dependovirus/genética , Saccharomyces cerevisiae/genética , Capsídeo , Proteínas do Capsídeo/metabolismo , DNA de Cadeia Simples/metabolismo , Expressão Gênica , Vetores Genéticos/genética , Vetores Genéticos/metabolismo , Humanos , Plasmídeos/genética , Plasmídeos/metabolismo , Saccharomyces cerevisiae/metabolismo
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