Glyco-engineered CHO cell lines producing alpha-1-antitrypsin and C1 esterase inhibitor with fully humanized N-glycosylation profiles.

Metabolic Engineering(2019)

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摘要
Recombinant Chinese hamster ovary (CHO) cells are able to provide biopharmaceuticals that are essentially free of human viruses and have N-glycosylation profiles similar, but not identical, to humans. Due to differences in N-glycan moieties, two members of the serpin superfamily, alpha-1-antitrypsin (A1AT) and plasma protease C1 inhibitor (C1INH), are currently derived from human plasma for treating A1AT and C1INH deficiency. Deriving therapeutic proteins from human plasma is generally a cost-intensive process and also harbors a risk of transmitting infectious particles. Recombinantly produced A1AT and C1INH (rhA1AT, rhC1INH) decorated with humanized N-glycans are therefore of clinical and commercial interest.
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关键词
A1AT,AATD,AUC,B3gnt2,Cas9,C1INH,CHO,CRISPR,FACS,FITC,Fut8,Glul,HAE,HM,indel,IVC,KO,mAb,Mgat4A,Mgat4B,Mgat5,MSX,sgRNA,SNA,Sppl3,St3gal3,St3gal4,St3gal6,ST6GAL1,VCD,WT
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