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    Amino-terminally truncated cystic fibrosis transmembrane conductance regulator
    3.
    发明授权
    Amino-terminally truncated cystic fibrosis transmembrane conductance regulator 失效
    氨基末端截短的囊性纤维化跨膜电导调节因子

    公开(公告)号：US5990279A

    公开(公告)日：1999-11-23

    申请号：US455552

    申请日：1995-05-31

    申请人： Barrie J. Carter ,  Terence Flotte ,  Sandra Afione ,  Rikki Solow

    发明人： Barrie J. Carter ,  Terence Flotte ,  Sandra Afione ,  Rikki Solow

    IPC分类号： A61K38/00 ,  A61K48/00 ,  C07K14/47 ,  C12N15/12 ,  C12N15/864 ,  C07K14/00 ,  C12P21/06

    CPC分类号： C12N15/86 ,  C07K14/4712 ,  A61K38/00 ,  A61K48/00 ,  C07K2319/00 ,  C12N2750/14143

    摘要： Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

    摘要翻译： 本文描述了包含修饰的腺相关病毒（AAV）DNA序列的重组DNA的构建，其能够用作用于使用包含AAV DNA的末端的新型转录启动子来表达外源DNA序列的真核表达载体。 显示可以从哺乳动物细胞中的该载体获得测试报告基因的表达。 进一步表明，载体和启动子的这种组合可用于引入和表达人基因并校正由突变体内源基因故障导致的人细胞遗传缺陷。 此外，载体可以用于通过表达由所述基因的一部分的融合和载体中包含的合成序列组成的人基因的修饰版本来校正遗传缺陷。

    Modified adeno-associated virus vector capable of expression from a novel promoter
    4.
    发明授权
    Modified adeno-associated virus vector capable of expression from a novel promoter 失效
    能够从新型启动子表达的修饰的腺相关病毒载体

    公开(公告)号：US6165781A

    公开(公告)日：2000-12-26

    申请号：US235375

    申请日：1999-01-21

    申请人： Barrie J. Carter ,  Terence Flotte ,  Sandra Afione ,  Rikki Solow

    发明人： Barrie J. Carter ,  Terence Flotte ,  Sandra Afione ,  Rikki Solow

    IPC分类号： A61K38/00 ,  A61K48/00 ,  C07K14/47 ,  C12N15/12 ,  C12N15/864 ,  C07H21/04

    CPC分类号： C12N15/86 ,  A61K38/00 ,  A61K48/00 ,  C07K14/4712 ,  C07K2319/00 ,  C12N2750/14143

    摘要： Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.

    摘要翻译： 本文描述了包含修饰的腺相关病毒（AAV）DNA序列的重组DNA的构建，其能够用作用于使用包含AAV DNA的末端的新型转录启动子来表达外源DNA序列的真核表达载体。 显示可以从哺乳动物细胞中的该载体获得测试报告基因的表达。 进一步表明，载体和启动子的这种组合可用于引入和表达人基因并校正由突变体内源基因故障导致的人细胞遗传缺陷。 此外，载体可以用于通过表达由所述基因的一部分的融合和载体中包含的合成序列组成的人基因的修饰版本来校正遗传缺陷。