AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice-Reference-Cited by-同舟云学术

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Published:2015-03-05 Issue:10 Volume:125 Page:1553-1561
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Crudele Julie M.¹,Finn Jonathan D.¹,Siner Joshua I.¹,Martin Nicholas B.¹,Niemeyer Glenn P.²,Zhou Shangzhen¹,Mingozzi Federico¹,Lothrop Clinton D.²,Arruda Valder R.¹³

Affiliation:

1. Department of Pediatrics, The Children’s Hospital of Philadelphia, Philadelphia, PA;

2. Department of Biochemistry, University of Alabama at Birmingham, Birmingham, AL; and

3. Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Abstract

Key Points Liver-restricted expression of FIX-Padua induces immune tolerance to the transgene in hemophilia B inhibitor dog models. Long-term toxicity studies show no increased risk of thrombogenicity of FIX-Padua in mice and dogs.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/125/10/1553/1382707/1553.pdf

Reference48 articles.

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3. Inhibitor in haemophilia B.;Warrier,2005

4. Inhibitors in congenital coagulation disorders.;Key;Br J Haematol,2004

5. Gene therapy for haemophilia: a long and winding road.;High;J Thromb Haemost,2011

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