发明公开
EP1497436A1 COMPOSITIONS FOR DELIVERING ENZYMES INVOLVED IN AMINO ACID METABOLISM USING RECOMBINANT ADENO-ASSOCIATED VIRUS VIRONS (RAAV VIRONS), AND METHOD AND USE FOR TREATING AMINO ACID METABOLIC DISORDERS USING SUCH RAAV VIRONS
有权
组成行政参与氨基酸代谢酶使用重组病毒腺相关病毒（rAAV病毒），和方法以及用于治疗氨基酸代谢紊乱使用这样的腺相关病毒的病毒粒子
- 专利标题： COMPOSITIONS FOR DELIVERING ENZYMES INVOLVED IN AMINO ACID METABOLISM USING RECOMBINANT ADENO-ASSOCIATED VIRUS VIRONS (RAAV VIRONS), AND METHOD AND USE FOR TREATING AMINO ACID METABOLIC DISORDERS USING SUCH RAAV VIRONS
- 专利标题（中）： 组成行政参与氨基酸代谢酶使用重组病毒腺相关病毒（rAAV病毒），和方法以及用于治疗氨基酸代谢紊乱使用这样的腺相关病毒的病毒粒子
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申请号： EP03717600.5申请日： 2003-04-16
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公开(公告)号： EP1497436A1公开(公告)日： 2005-01-19
- 发明人: Ozawa, Keiya , Mizukami, Hiroaki , Kume, Akihiro
- 申请人： Ozawa, Keiya , Mizukami, Hiroaki , Kume, Akihiro
- 申请人地址： Division of Genetic Therapeutics,Center for Molecular Medicine,Jichi Medical School,3311-1 Yakushiji,Minamikawachi-machi Kawachi-gun,Tochigi 329-0498 JP
- 专利权人： Ozawa, Keiya,Mizukami, Hiroaki,Kume, Akihiro
- 当前专利权人： Ozawa, Keiya,Mizukami, Hiroaki,Kume, Akihiro
- 当前专利权人地址： Division of Genetic Therapeutics,Center for Molecular Medicine,Jichi Medical School,3311-1 Yakushiji,Minamikawachi-machi Kawachi-gun,Tochigi 329-0498 JP
- 代理机构： Winter, Brandl, Fürniss, Hübner, Röss, Kaiser, PoltePartnerschaft
- 优先权： US124749 20020416
- 国际公布： WO2003087383 20031023
- 主分类号： C12N15/86
- IPC分类号： C12N15/86 ; A61K48/00
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. Methods for treating phenylketonuria are also described.
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