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Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies

Replication-defective (RD) recombinant simian virus 40 (SV40)-based gene delivery vectors hold a great potential for clinical applications because of their presumed non-immunogenicity and capacity to induce immune tolerance to the transgene products in humans. However, the clinical use of SV40 vecto...

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Autores principales: Toscano, Miguel G., van der Velden, Jeroen, van der Werf, Sybrand, Odijk, Machteld, Roque, Ana, Camacho-Garcia, Rafael J., Herrera-Gomez, Irene G., Mancini, Irene, de Haan, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537168/
https://www.ncbi.nlm.nih.gov/pubmed/28791314
http://dx.doi.org/10.1016/j.omtm.2017.06.007
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author Toscano, Miguel G.
van der Velden, Jeroen
van der Werf, Sybrand
Odijk, Machteld
Roque, Ana
Camacho-Garcia, Rafael J.
Herrera-Gomez, Irene G.
Mancini, Irene
de Haan, Peter
author_facet Toscano, Miguel G.
van der Velden, Jeroen
van der Werf, Sybrand
Odijk, Machteld
Roque, Ana
Camacho-Garcia, Rafael J.
Herrera-Gomez, Irene G.
Mancini, Irene
de Haan, Peter
author_sort Toscano, Miguel G.
collection PubMed
description Replication-defective (RD) recombinant simian virus 40 (SV40)-based gene delivery vectors hold a great potential for clinical applications because of their presumed non-immunogenicity and capacity to induce immune tolerance to the transgene products in humans. However, the clinical use of SV40 vectors has been hampered by the lack of a packaging cell line that produces replication-competent (RC) free SV40 particles in the vector production process. To solve this problem, we have adapted the current SV40 vector genome used for the production of vector particles and generated a novel Vero-based packaging cell line named SuperVero that exclusively expresses the SV40 large T antigen. SuperVero cells produce similar numbers of SV40 vector particles compared to the currently used packaging cell lines, albeit in the absence of contaminating RC SV40 particles. Our unique SV40 vector platform named SVac paves the way to clinically test a whole new generation of SV40-based therapeutics for a broad range of important diseases.
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spelling pubmed-55371682017-08-08 Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies Toscano, Miguel G. van der Velden, Jeroen van der Werf, Sybrand Odijk, Machteld Roque, Ana Camacho-Garcia, Rafael J. Herrera-Gomez, Irene G. Mancini, Irene de Haan, Peter Mol Ther Methods Clin Dev Original Article Replication-defective (RD) recombinant simian virus 40 (SV40)-based gene delivery vectors hold a great potential for clinical applications because of their presumed non-immunogenicity and capacity to induce immune tolerance to the transgene products in humans. However, the clinical use of SV40 vectors has been hampered by the lack of a packaging cell line that produces replication-competent (RC) free SV40 particles in the vector production process. To solve this problem, we have adapted the current SV40 vector genome used for the production of vector particles and generated a novel Vero-based packaging cell line named SuperVero that exclusively expresses the SV40 large T antigen. SuperVero cells produce similar numbers of SV40 vector particles compared to the currently used packaging cell lines, albeit in the absence of contaminating RC SV40 particles. Our unique SV40 vector platform named SVac paves the way to clinically test a whole new generation of SV40-based therapeutics for a broad range of important diseases. American Society of Gene & Cell Therapy 2017-07-05 /pmc/articles/PMC5537168/ /pubmed/28791314 http://dx.doi.org/10.1016/j.omtm.2017.06.007 Text en © 2017 The Authors http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Original Article
Toscano, Miguel G.
van der Velden, Jeroen
van der Werf, Sybrand
Odijk, Machteld
Roque, Ana
Camacho-Garcia, Rafael J.
Herrera-Gomez, Irene G.
Mancini, Irene
de Haan, Peter
Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
title Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
title_full Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
title_fullStr Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
title_full_unstemmed Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
title_short Generation of a Vero-Based Packaging Cell Line to Produce SV40 Gene Delivery Vectors for Use in Clinical Gene Therapy Studies
title_sort generation of a vero-based packaging cell line to produce sv40 gene delivery vectors for use in clinical gene therapy studies
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537168/
https://www.ncbi.nlm.nih.gov/pubmed/28791314
http://dx.doi.org/10.1016/j.omtm.2017.06.007
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