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发明公开
CN108103102A 一种AAV1病毒介导的骨骼肌特异性PCK1基因表达载体及其用途 有权 转让

一种AAV1病毒介导的骨骼肌特异性PCK1基因表达载体及其用途
摘要：
本发明提供一种肌肉组织特异性表达磷酸烯醇式丙酮酸羧激酶（phosphoenolpyruvatecarboxykinase,PEPCK）以降低高血脂、延缓衰老和提高生殖能力的基因表达和转移载体。所述载体含有改造后的人α‑骨骼肌肌动蛋白（α‑skeletal actin）基因的启动子、鼠细小病毒（minute virus of mice,MVM）的内含子、改造后的人PCK1（phosphoenolpyruvatecarboxykinase 1,PCK1）基因的cDNA和4个串联的人miR‑122靶序列。所述重组载体由腺相关病毒载体介导，包括但不限于1型腺相关病毒。
摘要（英）：

The invention provides a vector for expressing and transferring gene with muscle tissue specific expression of phosphoenolpyruvatecarboxykinase (PEPCK) for reducing hyperlipidemia, delaying aging, andimproving reproductive capacity. The vector contains a reconstructed promoter of human alpha-skeletal actin gene, an intron of minute virus of mice (MVM), reconstructed cDNA of human phosphoenolpyruvatecarboxykinase 1 (PCK1), and four human miR-122 target sequences which are connected in series connection. The recombinant vector is mediated by an adeno-associated virus vector, and comprises a type 1 adeno-associated virus and the like.

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