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dc.contributor.authorFreitas, Martiela Vaz dept_BR
dc.contributor.authorFrâncio, Larianept_BR
dc.contributor.authorHaleva, Laurapt_BR
dc.contributor.authorMatte, Ursula da Silveirapt_BR
dc.date.accessioned2024-03-28T06:25:17Zpt_BR
dc.date.issued2022pt_BR
dc.identifier.issn1415-4757pt_BR
dc.identifier.urihttp://hdl.handle.net/10183/274348pt_BR
dc.description.abstractThere are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the efficiency of gene therapy in different ways. Here we present an overview of approved gene therapy products and the immune response elicited by gene delivery systems. These include responses against the vector or its content after delivery and against the product of the corrected gene. Strategies to overcome the hurdles include hiding the vector or/and the transgene product from the immune system and hiding the immune system from the vector/transgene product. Combining different strategies, such as patient screening and intelligent vector design, gene therapy is set to make a difference in the life of patients with severe genetic diseases.en
dc.format.mimetypeapplication/pdfpt_BR
dc.language.isoengpt_BR
dc.relation.ispartofGenetics and molecular biology. Ribeirão Preto. Vol. 45, no. 3, supl. 1 (2022), e20220046 , 18 p.pt_BR
dc.rightsOpen Accessen
dc.subjectGene deliveryen
dc.subjectTerapia gênicapt_BR
dc.subjectResposta imunept_BR
dc.titleProtection is not always a good thing: the immune system’s impact on gene therapypt_BR
dc.typeArtigo de periódicopt_BR
dc.identifier.nrb001161965pt_BR
dc.type.originNacionalpt_BR


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