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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。

    Adeno-associated virus-mediated delivery of angiogenic factors
    7.
    发明公开
    Adeno-associated virus-mediated delivery of angiogenic factors 审中-公开
    Durch adenoassoziierte Viren vermittelte Freigabe von Angiogenesefaktoren

    公开(公告)号：EP1870473A1

    公开(公告)日：2007-12-26

    申请号：EP07010881.6

    申请日：2001-08-17

    申请人： Ozawa, Keiya ,  Shimpo, Masahisa ,  Ikeda, Uichi ,  Maeda, Yoshikazu ,  Shimada, Kazuyuki

    发明人： Ozawa, Keiya ,  Shimpo, Masahisa ,  Ikeda, Uichi ,  Maeda, Yoshikazu ,  Shimada, Kazuyuki

    IPC分类号： C12N15/864 ,  C12N15/12 ,  C07K14/50 ,  C07K14/52 ,  C07K14/515 ,  A61K48/00

    CPC分类号： A61K48/00 ,  A61K48/0075 ,  C07K14/52 ,  C12N15/86 ,  C12N2750/14143

    摘要： The use of recombinant adeno-associated virus (rAAV) virions for delivery of genes encoding angiogenic factors to muscle tissue is disclosed. The invention describes such methods of delivery and also describes methods for treating an ischemic condition such as myocardial ischemia. The methods include direct delivery of rAAV virions to a muscle via intramuscular injection and indirect delivery to a muscle via an injection into a blood vessel supplying blood to the muscle. The invention provides for sustained expression of the rAAV virion-delivered angiogenic factor gene or genes, which subsequently leads to a quantifiable therapeutic angiogenic factor gene or genes, which subsequently leads to a quantifiable therapeutic effect, namely an increase in new blood vessel formation resulting in an increase in blood flow to the ischemic muscle tissue.

    摘要翻译： 公开了使用重组腺相关病毒（rAAV）病毒颗粒将编码血管生成因子的基因递送至肌肉组织。 本发明描述了这种递送方法，并且还描述了治疗缺血状况如心肌缺血的方法。 所述方法包括通过肌内注射直接递送rAAV病毒体到肌肉，并通过注射到向肌肉供血的血管中间接递送至肌肉。 本发明提供rAAV病毒颗粒递送的血管发生因子基因或基因的持续表达，其随后导致可定量的治疗性血管生成因子基因或基因，其随后导致可定量的治疗效果，即增加新的血管形成，导致 血液流向缺血性肌肉组织的增加。