World Federation of Hemophilia Gene Therapy Registry

Author:

Konkle Barbara A.1,Coffin Donna2,Pierce Glenn F.2,Clark Cary3,George Lindsey45,Iorio Alfonso6,Mahlangu Johnny37,Naccache Mayss2,O’Mahony Brian89,Peyvandi Flora310,Pipe Steve1112,Quartel Adrian13,Sawyer Eileen K.14,Skinner Mark W.15,Tortella Bartholomew16,Watson Crystal17,Winburn Ian18,

Affiliation:

1. Bloodworks NWWashington Center for Bleeding Disorders Seattle WA USA

2. World Federation of Hemophilia Montreal QC Canada

3. International Society on Thrombosis and Hemostasis Carrboro NC USA

4. The Children's Hospital of Philadelphia Philadelphia PA USA

5. Univerisity of Pennsylvania Philadelphia PA USA

6. McMaster University Hamilton ON Canada

7. Haemophilia Comprehensive Care Centre University of the WitwatersrandNHLS and Charlotte Maxeke Johannesburg Academic Hospital Johannesburg South Africa

8. Irish Haemophilia Society Dublin Ireland

9. European Hemophilia Consortium Brussels Belgium

10. IRCCS Maggiore Hospital Milan and University of Milan Milan Italy

11. University of Michigan Pediatrics, Ann Arbor MI USA

12. National Hemophilia Foundation New York NY USA

13. BioMarin Pharmaceutical Inc Novato CA USA

14. uniQure Inc Lexington KY USA

15. Institute for Policy Advancement Ltd Washington DC USA

16. Spark Therapeutics Inc Philadelphia PA USA

17. American Thrombosis and Hemostssasis Network Chicago IL USA

18. Pfizer Inc New York NY USA

Publisher

Wiley

Subject

Genetics (clinical),Hematology,General Medicine

Reference17 articles.

1. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia

2. A Molecular Revolution in the Treatment of Hemophilia [published online ahead of print, 2019 Nov 13];Butterfield JSS;Mol Ther,2019

3. Gene therapy for hemophilia;Nathwani AC;Hematology Am Soc Hematol Educ Prog,2019

4. Characterization of the human factor VIII gene

5. Expression of active human factor VIII from recombinant DNA clones

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