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

MJ Osborn; A Panoskaltsis-Mortari; RT McElmurry; SK Bell; DAA Vignali; Martin Denis Ryan; A Wilbur; RS McIvor; J Tolar; BR Blazar

doi:10.1016/j.ymthe.2005.04.013

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

MJ Osborn, A Panoskaltsis-Mortari, RT McElmurry, SK Bell, DAA Vignali, Martin Denis Ryan, A Wilbur, RS McIvor, J Tolar, BR Blazar

Research output: Contribution to journal › Article › peer-review

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 language	English
Pages (from-to)	569-574
Number of pages	6
Journal	Molecular Therapy
Volume	12
DOIs	https://doi.org/10.1016/j.ymthe.2005.04.013
Publication status	Published - Sept 2005

Keywords

multicistronic
cis-acting hydrolase elements
internal ribosome entry site
iduronidase
mucopolysaccharidosis type I
whole body imaging
MOUTH-DISEASE VIRUS
ALPHA-L-IDURONIDASE
CLEAVAGE
POLYPROTEIN
PROGRESS
GENERATION
PROSPECTS
PROTEINS
CISTRON
HURLER

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10.1016/j.ymthe.2005.04.013

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Osborn, MJ., Panoskaltsis-Mortari, A., McElmurry, RT., Bell, SK., Vignali, DAA., Ryan, M. D., Wilbur, A., McIvor, RS., Tolar, J., & Blazar, BR. (2005). A picornaviral ‘2A-like’ sequence based tricistronic vector allowing for high level therapeutic gene expression coupled to a dual reporter system. Molecular Therapy, 12, 569-574. https://doi.org/10.1016/j.ymthe.2005.04.013

@article{94912d24a4ec4d32a43cfc9b132ec88b,

title = "A picornaviral {\textquoteleft}2A-like{\textquoteright} sequence based tricistronic vector allowing for high level therapeutic gene expression coupled to a dual reporter system.",

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.",

keywords = "multicistronic, cis-acting hydrolase elements, internal ribosome entry site, iduronidase, mucopolysaccharidosis type I, whole body imaging, MOUTH-DISEASE VIRUS, ALPHA-L-IDURONIDASE, CLEAVAGE, POLYPROTEIN, PROGRESS, GENERATION, PROSPECTS, PROTEINS, CISTRON, HURLER",

author = "MJ Osborn and A Panoskaltsis-Mortari and RT McElmurry and SK Bell and DAA Vignali and Ryan, {Martin Denis} and A Wilbur and RS McIvor and J Tolar and BR Blazar",

year = "2005",

month = sep,

doi = "10.1016/j.ymthe.2005.04.013",

language = "English",

volume = "12",

pages = "569--574",

journal = "Molecular Therapy",

issn = "1525-0016",

publisher = "Nature publishing group",

}

Osborn, MJ, Panoskaltsis-Mortari, A, McElmurry, RT, Bell, SK, Vignali, DAA, Ryan, MD, Wilbur, A, McIvor, RS, Tolar, J & Blazar, BR 2005, 'A picornaviral ‘2A-like’ sequence based tricistronic vector allowing for high level therapeutic gene expression coupled to a dual reporter system.', Molecular Therapy, vol. 12, pp. 569-574. https://doi.org/10.1016/j.ymthe.2005.04.013

TY - JOUR

T1 - A picornaviral ‘2A-like’ sequence based tricistronic vector allowing for high level therapeutic gene expression coupled to a dual reporter system.

AU - Osborn, MJ

AU - Panoskaltsis-Mortari, A

AU - McElmurry, RT

AU - Bell, SK

AU - Vignali, DAA

AU - Ryan, Martin Denis

AU - Wilbur, A

AU - McIvor, RS

AU - Tolar, J

AU - Blazar, BR

PY - 2005/9

Y1 - 2005/9

N2 - 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.

AB - 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.

KW - multicistronic

KW - cis-acting hydrolase elements

KW - internal ribosome entry site

KW - iduronidase

KW - mucopolysaccharidosis type I

KW - whole body imaging

KW - MOUTH-DISEASE VIRUS

KW - ALPHA-L-IDURONIDASE

KW - CLEAVAGE

KW - POLYPROTEIN

KW - PROGRESS

KW - GENERATION

KW - PROSPECTS

KW - PROTEINS

KW - CISTRON

KW - HURLER

UR - http://www.scopus.com/inward/record.url?scp=23844466445&partnerID=8YFLogxK

U2 - 10.1016/j.ymthe.2005.04.013

DO - 10.1016/j.ymthe.2005.04.013

M3 - Article

SN - 1525-0016

VL - 12

SP - 569

EP - 574

JO - Molecular Therapy

JF - Molecular Therapy

ER -

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

Abstract

Keywords

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