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GENETICALLY MODIFIED PROBIOTIC FOR THE TREATMENT OF PHENYLKETONURIA (PKU) DISEASE

机译:遗传修饰的脯氨酸治疗苯丙酮尿症(PKU)疾病

摘要

A GMP adapted to provide the PAL gene for the treatment of PKU when administered orally. The GMP of the present invention may include a probiotic, a PAL gene to be expressed using the probiotic, wherein the PAL gene is functionally attached to a promoter and a ribosome binding site, and may be codon-optimized for expression in a certain host organism. A method of treating the metabolic disease of PKU by oral administration and ingestion of a GMP is also provided.
机译:口服时适于提供PAL基因以治疗PKU的GMP。本发明的GMP可以包括益生菌,使用该益生菌表达的PAL基因,其中该PAL基因在功能上附着于启动子和核糖体结合位点,并且可以被密码子优化以在某些宿主生物中表达。 。还提供了一种通过口服给药和摄入GMP治疗PKU代谢疾病的方法。

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