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EXTRACELLULAR VESICLES FOR REPLACEMENT OF UREA CYCLE PROTEINS NUCLEIC ACIDS
EXTRACELLULAR VESICLES FOR REPLACEMENT OF UREA CYCLE PROTEINS NUCLEIC ACIDS
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机译:用于更换尿素循环蛋白和核酸的细胞外囊泡
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摘要
The present invention relates to engineered extracellular vesicles (EVs) as a novel therapeutic approach to treating urea cycle disorders. More specifically, the invention relates to the use of various protein engineering and nucleic acid engineering strategies for improving loading of urea cycle proteins or nucleic acids encoding urea cycle proteins into EVs and targeting of the resultant EVs to tissues and organs of interest.
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