Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B-Reference-Cited by-同舟云学术

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Published:2018-03-01 Issue:9 Volume:131 Page:1022-1031
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Miesbach Wolfgang¹^ORCID,Meijer Karina²,Coppens Michiel³,Kampmann Peter⁴,Klamroth Robert⁵,Schutgens Roger⁶,Tangelder Marco⁷,Castaman Giancarlo⁸,Schwäble Joachim⁹,Bonig Halvard⁹¹⁰,Seifried Erhard⁹,Cattaneo Federica¹¹,Meyer Christian⁷,Leebeek Frank W. G.¹²

Affiliation:

1. University Hospital Frankfurt, Frankfurt, Germany;

2. University Medical Centre Groningen, University of Groningen, Groningen, The Netherlands;

3. Academic Medical Center, Amsterdam, The Netherlands;

4. Rigshospitalet, Copenhagen, Denmark;

5. Vivantes Klinikum, Berlin, Germany;

6. University Medical Centre Utrecht and University Utrecht, Utrecht, The Netherlands;

7. uniQure biopharma B.V., Amsterdam, The Netherlands;

8. Azienda Ospedaliera Universitaria Careggi, Florence, Italy;

9. Institute for Transfusion Medicine and Immunohematology, Goethe University Hospital Medical School, German Red Cross Blood Donor Service, Frankfurt, Germany;

10. Department of Medicine/Hematology, University of Washington, Seattle, WA;

11. Chiesi Farmaceutici S.p.A., Parma, Italy; and

12. Erasmus University Medical Centre, Rotterdam, The Netherlands

Abstract

Key Points AAV5 liver-directed wild-type hFIX gene transfer was well tolerated and clinically effective in severe and moderate-severe hemophilia B. No cellular immune responses to the AAV5 vector were detected, and FIX expression levels were stable for the entire observation period.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/131/9/1022/1406467/blood804419.pdf

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3. World Federation of Hemophilia. Report on the Annual Global Survey2010. https://www1.wfh.org/publication/files/pdf-1427.pdf. Accessed 25 May 2017.

4. Musculoskeletal complications of hemophilia;Rodriguez-Merchan;HSS J,2010

5. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia;Manco-Johnson;N Engl J Med,2007

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