GENE THERAPY FOR TREATING PHENYLKETONURIA
Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase. 提供了可用于治疗苯丙酮尿症的组合物和方案。所述组合物包含具有驱动人类苯丙氨酸羟化酶表达的转甲状腺素蛋白增强子和启动子...
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| Main Authors | , , |
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| Format | Patent |
| Language | Chinese English |
| Published |
11.10.2019
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| Subjects | |
| Online Access | Get full text |
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| Abstract | Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.
提供了可用于治疗苯丙酮尿症的组合物和方案。所述组合物包含具有驱动人类苯丙氨酸羟化酶表达的转甲状腺素蛋白增强子和启动子的重组腺相关病毒(rAAV)。 |
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| AbstractList | Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.
提供了可用于治疗苯丙酮尿症的组合物和方案。所述组合物包含具有驱动人类苯丙氨酸羟化酶表达的转甲状腺素蛋白增强子和启动子的重组腺相关病毒(rAAV)。 |
| Author | WILSON JAMES M ASHLEY SCOTT SIDRANE JENNY AGNES |
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| DocumentTitleAlternate | 用于治疗苯丙酮尿症的基因疗法 |
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| Snippet | Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a... |
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| Title | GENE THERAPY FOR TREATING PHENYLKETONURIA |
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