A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system

Mark J. Osborn, Angela Panoskaltsis-Mortari, Ron T. McElmurry, Scott K. Bell, Dario A A Vignali, Martin D. Ryan, Andrew C. Wilber, R. Scott McIvor, Jakub Tolar, Bruce R. Blazar

Research output: Contribution to journalArticle

53 Scopus citations

Abstract

The 2A-like sequences from members of the picornavirus family were utilized to construct a tricistronic vector bearing the human iduronidase (IDUA) gene along with the firefly luciferase and DsRed2 reporter genes. The 2A-like sequences mediate a cotranslational cleavage event resulting in the release of each individual protein product. Efficient cleavage was observed and all three proteins were functional in vitro and in vivo, allowing for supratherapeutic IDUA enzyme levels and the coexpression of luciferase and DsRed2 expression, which enabled us to track gene expression.

Original languageEnglish (US)
Pages (from-to)569-574
Number of pages6
JournalMolecular Therapy
Volume12
Issue number3
DOIs
StatePublished - Sep 1 2005

Keywords

  • Cis-acting hydrolase elements
  • Iduronidase
  • Internal ribosome entry site
  • Mucopolysaccharidosis type I
  • Multicistronic
  • Whole body imaging

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