Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

New England Journal of Medicine - Tập 365 Số 25 - Trang 2357-2365 - 2011
Amit C. Nathwani1,2,3, Graham Davies2, Savita Rangarajan4, Cecilia Rosales1,3, Jenny McIntosh1,3, David C. Linch1, Pratima Chowdary2, Anne Riddell2, Arnulfo Pie2, Chris F. Harrington2, James O’Beirne5, Keith Smith3, John Pasi6, Bertil Glader7, Pradip K. Rustagi7, Catherine Y. Ng8, Mark A. Kay7, Jianli Zhou8, Yunyu Spence8, Christopher L. Morton8, James A. Allay9, John B. Coleman9, Susan Sleep9, John M. Cunningham10, Deo Kumar Srivastava11, Etiena Basner‐Tschakarjan12, Federico Mingozzi12, Katherine A. High12,13, John T. Gray14, Ulrike M. Reiss14, Arthur W. Nienhuis14, Andrew M. Davidoff8
1Department of Haematology, University College London Cancer Institute, Trust
2Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Trust
3NHS Blood and Transplant
4Basingstoke and North Hampshire NHS Foundation Trust, Basingstoke, United Kingdom, St. Jude Children's Research Hospital
5Liver Unit, Trust
6Centre for Haematology, Barts and the London, Queen Mary's School of Medicine
7Stanford University School of Medicine, Palo Alto, CA, St. Jude Children's Research Hospital
8Departments of Surgery, St. Jude Children's Research Hospital
9Children's GMP
10Department of Pediatrics, University of Chicago, Chicago
11Biostatistics, St. Jude Children's Research Hospital
12Center for Cellular and Molecular Therapeutics at Children's Hospital of Philadelphia, Philadelphia
13Howard Hughes Medical Institute, Chevy Chase, MD
14Hematology, St. Jude Children's Research Hospital

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