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Increase of the expression levels of factor VIII by insertion of spliceable nucleotide sequences into factor VIII cDNA

机译:通过将可剪接的核苷酸序列插入因子VIII cDNA中来增加因子VIII的表达水平

摘要

A modified factor VIII cDNA is disclosed, wherein in introns 1 and/or 13 of the wild-type factor VIII cDNA one or more splicable nucleotide sequences or a nucleotide sequence which will be spliced during the export of the pre-mRNA from the nucleus have been inserted. Further a process for the production of a biologically active recombinant human factor VIII or its derivative is disclosed, which is performed by cultivating an animal cell line comprising a recombinant expression vector containing said modified factor VIII cDNA. Moreover a transfer vector for use in the human gene therapy is described which comprises said modified factor VIII cDNA.
机译:公开了一种修饰的因子VIII cDNA,其中在野生型因子VIII cDNA的内含子1和/或13中,一个或多个可剪接的核苷酸序列或在从细胞核输出前mRNA的过程中将被剪接的核苷酸序列具有一个或多个。被插入。进一步公开了生产生物活性重组人因子VIII或其衍生物的方法,该方法通过培养动物细胞系来进行,所述动物细胞系包含含有所述修饰的因子VIII cDNA的重组表达载体。此外,描述了用于人类基因治疗的转移载体,其包含所述修饰的因子VIII cDNA。

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