期刊
MOLECULAR THERAPY
卷 12, 期 3, 页码 569-574出版社
NATURE PUBLISHING GROUP
DOI: 10.1016/j.ymthe.2005.04.013
关键词
multicistronic; cis-acting hydrolase elements; internal ribosome entry site; iduronidase; mucopolysaccharidosis type I; whole body imaging
资金
- NHLBI NIH HHS [R01 HL49997, HL52592] Funding Source: Medline
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.
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