发明名称 |
Methods of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
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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申请公布号 |
US2005147592(A1) |
申请公布日期 |
2005.07.07 |
申请号 |
US20040992346 |
申请日期 |
2004.11.18 |
申请人 |
OZAWA KEIYA;MIZUKAMI HIROAKI;KUME AKIHIRO |
发明人 |
OZAWA KEIYA;MIZUKAMI HIROAKI;KUME AKIHIRO |
分类号 |
C12N15/09;A61K35/76;A61K38/43;A61K38/44;A61K47/48;A61K48/00;A61P3/00;A61P9/00;A61P9/04;A61P43/00;C12N15/864;(IPC1-7):A61K48/00;C12N15/861 |
主分类号 |
C12N15/09 |
代理机构 |
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