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The effect of a secretion-enhanced heavy chain on improving intein-based dual-vector co-delivery of a full-length factor VIII gene

Autor: Xiao-Yan Chi, Fu-Xiang Zhu, Hui-Ge Qu, Jing Miao, Ze-Long Liu, Shu-De Yang

Publikováno v: Chinese Science Bulletin. 56:158-163

Treatment of hemophilia A by gene therapy is adversely affected by inefficient FVIII secretion and the large FVIII gene, which is difficult to package in the promising adeno-associated virus (AAV) vectors. Inhibited secretion of FVIII is caused mainl

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::13458bb440c63f1c4c4ba6543e94bf30
https://doi.org/10.1007/s11434-010-4244-7

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[Trans-splicing of Cys mutated coagulation factor VIII]

Autor: Fu-Xiang, Zhu, Ze-Long, Liu, Jing, Miao, Hui-Ge, Qu, Xiao-Yan, Chi

Publikováno v: Yao xue xue bao = Acta pharmaceutica Sinica. 47(6)

To investigate the improving effect of inter-chain disulfide formation on protein trans-splicing, we introduce a Cys point mutation at Tyr(664) in heavy chain and at Thr(1826) in light chain of B-domain-deleted FVIII (BDD-FVIII). By co-transfection o

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::20a9520ca961b69dcae365ca5609ca2b
https://pubmed.ncbi.nlm.nih.gov/22919720

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Inter-chain disulfide bond improved protein trans-splicing increases plasma coagulation activity in C57BL/6 mice following portal vein FVIII gene delivery by dual vectors

Autor: Xiao-Yan Chi, Hui-Ge Qu, Jing Miao, XiaoLei Wang, Fu-Xiang Zhu, Ze-Long Liu

Publikováno v: Science China. Life sciences. 56(3)

Protein trans-splicing based dual-vector factor VIII (FVIII) gene delivery is adversely affected by less efficiency of protein splicing. We sought to increase the amount of spliced FVIII protein and plasma coagulation activity in dual-vector FVIII tr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::51337193acaf90334eba933d398b6913
https://pubmed.ncbi.nlm.nih.gov/23526393

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[Leucine zippers improves protein splicing-mediated coagulation factor VIII gene delivery by dual-vector system]

Autor: Fu-Xiang, Zhu, Shu-De, Yang, Ze-Long, Liu, Jing, Miao, Hui-Ge, Qu, Xiao-Yan, Chi

Publikováno v: Yao xue xue bao = Acta pharmaceutica Sinica. 47(1)

In our recent study by exploring an intein-based dual-vector to deliver a B-domain-deleted FVIII (BDD-FVIII) gene, it showed that covalently ligated intact BDD-FVIII molecules with a specific coagulant activity could be produced from expressed heavy

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::fcdfcada7b17b97685af34e32cec80d0
https://pubmed.ncbi.nlm.nih.gov/22493803

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[Enhancing effect of deoxynivalenol-mediated GRP78 down-regulation on heavy chain secretion and bioactivity of two-chain FVIII gene co-transfected cells]

Autor: Fu-Xiang, Zhu, Shu-De, Yang, Ze-Long, Liu, Jing, Miao, Hui-Ge, Qu, Xiao-Yan, Chi

Publikováno v: Yao xue xue bao = Acta pharmaceutica Sinica. 46(12)

Although two chain transfering separately could be used to overcome the volume limitation of adeno-associated virus vectors (AAV) in coagulation factor VIII (FVIII) gene delivery, it leads to chain imbalance for inefficient heavy chain secretion. In

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::4b9d0885db4f45c2f9d9fc1fbcb6b4fa
https://pubmed.ncbi.nlm.nih.gov/22375418

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[Glycosylation and L303e/F309S mutations improve intein-mediated splicing of the split coagulation factor VIII]

Autor: Fu-Xiang, Zhu, Ze-Long, Liu, Jing, Miao, Hui-Ge, Qu, Xiao-Yan, Chi

Publikováno v: Yao xue xue bao = Acta pharmaceutica Sinica. 45(11)

We recently demonstrated that an intein-mediated protein splicing can be used to transfer B-domain-deleted FVIII (BDD-FVIII) gene by a dual-vector. In this study, we observed the effect of a variant heavy chain with six potential glycosylation sites

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::33e4dfe2899f4c1fea05ddf09c75ebf6
https://pubmed.ncbi.nlm.nih.gov/21361035

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[Post-translational ligation and function of dual-vector transferred split CFTR gene]

Autor: Fu-Xiang, Zhu, Ze-Long, Liu, Hui-Ge, Qu, Xiao-Yan, Chi

Publikováno v: Yao xue xue bao = Acta pharmaceutica Sinica. 45(1)

The mutation of cystic fibrosis transmembrane conductance regulator (CFTR) gene leads to an autosomal recessive genetic disorder cystic fibrosis (CF). The gene therapy for CF using adeno-associated virus (AAV) vectors delivering CFTR gene is restrict

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::75c4f48b967fda6df44ffb549b2318e8
https://pubmed.ncbi.nlm.nih.gov/21351451

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[Enhancing effect of AR-3 on protein splicing-based eukaryotic cell gene transfer of full-length coagulation factor VIII]

Autor: Fu-Xiang, Zhu, Ze-Long, Liu, Jing, Miao, Hui-Ge, Qu, Xiao-Yan, Chi

Publikováno v: Zhonghua yi xue za zhi. 90(48)

To study the effect of an acidic region-3 (AR-3), capable of improving the secretion of heavy chain of coagulation factor VIII (fVIII), on the secretion of protein spicing ligated full-length fVIII.A pair of vectors was used to deliver intein fused h

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::61339d8b919ac8dd3d3fb5851bccc29d
https://pubmed.ncbi.nlm.nih.gov/21223821

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