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GENETICALLY MODIFIED PROBIOTIC FOR THE TREATMENT OF PHENYLKETONURIA (PKU) DISEASE
GENETICALLY MODIFIED PROBIOTIC FOR THE TREATMENT OF PHENYLKETONURIA (PKU) DISEASE
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机译:遗传修饰的脯氨酸治疗苯丙酮尿症(PKU)疾病
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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.
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