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

作者信息

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

机构信息

Pediatrics, Hematology-Oncology, Blood and Marrow Transplant Program, Institute of Human Genetics, Department of Genetics, Cell Biology, and Development, University of Minnesota, Minneapolis, MN 55455, USA.

出版信息

Mol Ther. 2005 Sep;12(3):569-74. doi: 10.1016/j.ymthe.2005.04.013.

DOI:10.1016/j.ymthe.2005.04.013
PMID:15964244
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.

摘要

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