Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A-Reference-Cited by-同舟云学术

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Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

Published:2009-07 Issue:7 Volume:17 Page:1145-1154
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Doering Christopher B,Denning Gabriela,Dooriss Kerry,Gangadharan Bagirath,Johnston Jennifer M,Kerstann Keith W,McCarty David A,Spencer H Trent

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference34 articles.

1. Gene therapy for haemophilia;Murphy;Br J Haematol,2008

2. High-level expression of recombinant porcine coagulation factor VIII;Doering;J.Biol.Chem,2002

3. Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion;Doering;J Biol Chem,2004

4. Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity;Doering;Mol Ther,2007

5. High-level expression of porcine factor VIII from genetically modified bone marrow-derived stem cells;Gangadharan;Blood,2006

Cited by 56 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Humanization and functional characterization of enhanced coagulation factor IX variants identified through ancestral sequence reconstruction;Journal of Thrombosis and Haemostasis;2023-11

2. Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice;Journal of Thrombosis and Haemostasis;2023-08

3. Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII;Frontiers in Immunology;2022-12-15

4. Structural insights into blood coagulation factor VIII: Procoagulant complexes, membrane binding, and antibody inhibition;Journal of Thrombosis and Haemostasis;2022-09

5. Pharmacokinetic analysis identifies a factor VIII immunogenicity threshold after AAV gene therapy in hemophilia A mice;Blood Advances;2022-04-22

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