Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide–based retroviral vector View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2004-05

AUTHORS

Andrea L Szymczak, Creg J Workman, Yao Wang, Kate M Vignali, Smaroula Dilioglou, Elio F Vanin, Dario A A Vignali

ABSTRACT

Attempts to generate reliable and versatile vectors for gene therapy and biomedical research that express multiple genes have met with limited success. Here we used Picornavirus 'self-cleaving' 2A peptides, or 2A-like sequences from other viruses, to generate multicistronic retroviral vectors with efficient translation of four cistrons. Using the T-cell receptor:CD3 complex as a test system, we show that a single 2A peptide-linked retroviral vector can be used to generate all four CD3 proteins (CD3epsilon, gamma, delta, zeta), and restore T-cell development and function in CD3-deficient mice. We also show complete 2A peptide-mediated 'cleavage' and stoichiometric production of two fluorescent proteins using a fluorescence resonance energy transfer-based system in multiple cell types including blood, thymus, spleen, bone marrow and early stem cell progenitors. More... »

PAGES

589-594

Journal

TITLE

Nature Biotechnology

ISSUE

5

VOLUME

22

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/nbt957

    DOI

    http://dx.doi.org/10.1038/nbt957

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1039129492

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/15064769


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