Dual Vectors Expressing Murine Factor VIII Result in Sustained Correction of Hemophilia A Mice-Reference-Cited by-同舟云学术

Dual Vectors Expressing Murine Factor VIII Result in Sustained Correction of Hemophilia A Mice

Published:2003-01-15 Issue:2 Volume:14 Page:143-152
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Mah Cathryn¹,Sarkar Rita²,Zolotukhin Irene¹,Schleissing Mary¹,Xiao Xiao³,Kazazian Haig H.²,Byrne Barry J.¹

Affiliation:

1. Department of Pediatrics, Department of Molecular Genetics and Microbiology, and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610

2. Department of Genetics, University of Pennsylvania School of Medicine, Philadelphia, PA 19104

3. Department of Molecular Genetics and Biochemistry, Gene Therapy Center, and Duchenne Muscular Dystrophy Research Center, University of Pittsburgh, Pittsburgh, PA 15261

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/104303403321070838

Reference50 articles.

1. Molecular etiology of factor VIII deficiency in hemophilia A

2. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector

3. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A

4. Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies

5. Adenovirus-Mediated Factor VIII Gene Expression Results in Attenuated Anti-Factor VIII-Specific Immunity in Hemophilia A Mice Compared with Factor VIII Protein Infusion

Cited by 32 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. Long-term correction of hemorrhagic diathesis in hemophilia A mice by an AAV-delivered hybrid FVIII composed of the human heavy chain and the rat light chain;Frontiers of Medicine;2022-01-17

2. Guest Editor: G. Castaman GENE THERAPY FOR HEMOPHILIA: FACTS AND QUANDARIES IN THE 21ST CENTURY;Mediterranean Journal of Hematology and Infectious Diseases;2020-08-29

3. Evaluation of the activity levels of rat FVIII and human FVIII delivered by adeno-associated viral vectors both in vitro and in vivo;Blood Cells, Molecules, and Diseases;2018-11

4. Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects;Journal of Inherited Metabolic Disease;2017-05-31

5. MicroRNA-511 Binds to FKBP5 mRNA, Which Encodes a Chaperone Protein, and Regulates Neuronal Differentiation;Journal of Biological Chemistry;2016-08