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    Compositions for delivering enzymes involved in amino acid metabolism using recombinant adeno-associated virus virions (raav virions), and method and use for treating amino acid metabolic disorders using such raav virions
    2.
    发明公开
    Compositions for delivering enzymes involved in amino acid metabolism using recombinant adeno-associated virus virions (raav virions), and method and use for treating amino acid metabolic disorders using such raav virions 审中-公开
    组合物用于通过使用重组腺相关病毒病毒体（rAAV病毒）的参与氨基酸代谢的酶的施用和的方法和用于使用此类rAAV病毒治疗氨基酸代谢紊乱

    公开(公告)号：EP1847614A1

    公开(公告)日：2007-10-24

    申请号：EP07013616.3

    申请日：2003-04-16

    申请人： Ozawa, Keiya ,  Mizukami, Hiroaki ,  Kume, Akihiro

    发明人： Ozawa, Keiya ,  Mizukami, Hiroaki ,  Kume, Akihiro

    IPC分类号： C12N15/86 ,  A61K48/00

    CPC分类号： C12N15/86 ,  A61K38/44 ,  A61K47/6901 ,  A61K48/0075 ,  C12N2750/14143 ,  C12Y114/16001

    摘要： Methods for delivering a heterologous gene to a mammalian subject using recombinant adeno-associated virus (rAAV) virions are described. Recombinant AAV virions containing a heterologous gene encoding a metabolic protein are delivered to a mammalian subject having a metabolic disorder. The rAAV virion-delivered heterologous gene is expressed at a therapeutic level thereby ameliorating a sign or symptom of the metabolic disease. Exemplary examples of metabolic diseases are those caused by defects in aromatic amino acid metabolism. Exemplary examples of heterologous genes include those encoding an aromatic amino acid hydroxylase, aromatic amino acid decarboxylase, and enzymes involved in tetrahydrobiopterin synthesis.

    摘要翻译： 使用重组腺相关病毒递送异源基因到哺乳动物受试者的方法（的rAAV）病毒体的描述。 重组AAV病毒体包含编码蛋白质代谢的异源基因递送至患有代谢紊乱的哺乳动物受试者。 所述的rAAV病毒体递送异源基因在治疗水平由此改善所述代谢性疾病的体征或症状是过表达的。 代谢性疾病的示例性实例是那些通过在芳族氨基酸代谢缺陷引起的。 异源基因的示例性实例包括芳香族氨基酸羟化酶那些编码的芳族氨基酸脱羧酶，和在四氢合成有关的酶。