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    • 9. 发明申请
    • EXPRESSION CASSETTE FOR PERSISTENCE OF EXPRESSION OF A GENE OF INTEREST IN MUSCLE CELLS
    • 在肌肉细胞中表达兴趣基因的表达表达的表达
    • WO2003074711A1
    • 2003-09-12
    • PCT/IB2003/001360
    • 2003-03-04
    • TRANSGENE S.A.
    • ANTONIOU, MichaelRAGUZ, SelinaJACOBS, EricSILVESTRE, Nathalie
    • C12N15/67
    • C07K14/4708A61K48/00C12N2799/022
    • The present invention concerns the use of a nucleic acid fragment comprising a portion of at least 100 contigous nucleotide bases which portion has a sequence the same as, or homologous to a portion of corresponding length of the sequence as set out in SEQ ID NO: 1 starting at nucleotide approximately 1 and ending at nucleotide approximately 16879 or the same as, or homologous to a portion of the corresponding length of the sequence complementary to said SEQ ID NO: 1 within the specified positions, for improving the persistence of transfected cells expressing one or more gene(s) of interest operably linked to said acid nucleic fragment. The invention also relates to an expression cassette for the expression of one or more gene(s) of interest which expression is controlled by one or more control sequence(s) and a nucleic acid fragment as defined above. The present invention also provides a vector containing said expression cassette and a eukaryotic host cell containing said expression cassette or vector as well as a 15 composition comprising said expression cassette, vector or eukaryotic host cell for therapeutic or prophylactic purposes. The present invention is useful for many applications including the the construction of transgenic animal models and somatic gehe therapy, especially for treating or preventing muscle-affecting diseases.
    • 本发明涉及包含至少100个连续核苷酸碱基的一部分的核酸片段的用途,该部分具有与SEQ ID NO:1所示的序列的相应长度的一部分相同或同源的序列 从核苷酸大约1开始,以与指定位置内与所述SEQ ID NO:1互补的序列的相应长度的一部分相同或相同的核苷酸为16879,以改善表达一个的转染细胞的持续性 或更多的可操作地连接到所述酸性核酸片段的基因。 本发明还涉及用于表达一种或多种感兴趣的基因的表达盒,其表达受一个或多个控制序列和如上定义的核酸片段的控制。 本发明还提供含有所述表达盒和含有所述表达盒或载体的真核宿主细胞的载体以及包含所述表达盒,载体或真核宿主细胞用于治疗或预防目的的组合物。 本发明可用于许多应用,包括构建转基因动物模型和体细胞地球疗法,特别是用于治疗或预防肌肉疾病的疾病。