GENE THERAPY FOR TREATING PHENYLKETONURIA

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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Hauptverfasser: SIDRANE JENNY AGNES, WILSON JAMES M, ASHLEY SCOTT
Format: Patent
Sprache:chi ; eng
Schlagworte:
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Zusammenfassung: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)。