Rescue of Adeno-Associated Virus Production by shRNA Cotransfection

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dc.contributor.authorDiniz, Maria Cambraia Guimaro
dc.contributor.authorAfione, Sandra A.
dc.contributor.authorTanaka, Tsutomu
dc.contributor.authorChiorini, John A.
dc.date.accessioned2022-10-31T14:57:03Z
dc.date.available2022-10-31T14:57:03Z
dc.date.issued2020-10-16
dc.description.abstractAdeno-associated virus (AAV) vector technology is rapidly advancing and becoming not only the leading vector platform in the field of gene therapy but also a useful tool for functional genomic studies of novel proteins. As most vectors utilize constitutive promoters, this results in transgene expression during production. Depending on the transgene product, this could induce proapoptotic, cytostatic, or other unknown effects that interfere with producer cell function and, therefore, reduce viral vector yield. This can be a major limitation when trying to characterize poorly described genes. We describe the novel use of shRNA encoding plasmids cotransfected during packaging to limit the expression of the cytotoxic transgene product. This allowed the production of an otherwise unpackageable vector. The approach is simple, versatile, does not require modification of the vector plasmid, and should be easily adaptable to almost any transgene with minimal cost.en_US
dc.description.sponsorshipThis study was supported by an NIDCR, NIH intramural research grant to J.A.C.en_US
dc.description.urihttps://www.liebertpub.com/doi/epub/10.1089/hum.2019.249en_US
dc.format.extent6 pagesen_US
dc.genrejournal articlesen_US
dc.identifierdoi:10.13016/m2boes-fh3r
dc.identifier.citationGuimaro MC, Afione SA, Tanaka T, Chiorini JA. Rescue of Adeno-Associated Virus Production by shRNA Cotransfection. Hum Gene Ther. 2020;31(19-20):1068-1073. doi:10.1089/hum.2019.249en_US
dc.identifier.urihttps://doi.org/10.1089/hum.2019.249
dc.identifier.urihttp://hdl.handle.net/11603/26241
dc.language.isoen_USen_US
dc.publisherMary Ann Lieberten_US
dc.relation.isAvailableAtThe University of Maryland, Baltimore County (UMBC)
dc.relation.ispartofUMBC Office of the Dean of the College of Natural and Mathematical Sciences
dc.relation.ispartofUMBC Faculty Collection
dc.rightsThis work was written as part of one of the author's official duties as an Employee of the United States Government and is therefore a work of the United States Government. In accordance with 17 U.S.C. 105, no copyright protection is available for such works under U.S. Law.en_US
dc.rightsPublic Domain Mark 1.0*
dc.rights.urihttp://creativecommons.org/publicdomain/mark/1.0/*
dc.titleRescue of Adeno-Associated Virus Production by shRNA Cotransfectionen_US
dc.typeTexten_US
dcterms.creatorhttps://orcid.org/0000-0002-2633-9710en_US

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