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| Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector MA Kay, CS Manno, MV Ragni, PJ Larson, LB Couto, A McClelland, ... Nature genetics 24 (3), 257-261, 2000 | 1325 | 2000 |
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| Hemophilia B gene therapy with a high-specific-activity factor IX variant LA George, SK Sullivan, A Giermasz, JEJ Rasko, BJ Samelson-Jones, ... New England Journal of Medicine 377 (23), 2215-2227, 2017 | 738 | 2017 |
| Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer F Mingozzi, YL Liu, E Dobrzynski, A Kaufhold, JH Liu, YQ Wang, ... The Journal of clinical investigation 111 (9), 1347-1356, 2003 | 554 | 2003 |
| Combined effect of factor V Leiden and prothrombin 20210A on the risk of venous thromboembolism J Emmerich, FR Rosendaal, M Cattaneo, M Margaglione, V De Stefano, ... Thrombosis and haemostasis 86 (09), 809-816, 2001 | 545 | 2001 |
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| Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy JD Mount, RW Herzog, DM Tillson, SA Goodman, N Robinson, ... Blood, The Journal of the American Society of Hematology 99 (8), 2670-2676, 2002 | 449 | 2002 |
| The mutation Ala677→ Val in the methylene tetrahydrofolate reductase gene: a risk factor for arterial disease and venous thrombosis VR Arruda, PM von Zuben, LC Chiaparini, JM Annichino-Bizzacchi, ... Thrombosis and haemostasis 77 (05), 0818-0821, 1997 | 412 | 1997 |
| Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy GP Niemeyer, RW Herzog, J Mount, VR Arruda, DM Tillson, J Hathcock, ... Blood, The Journal of the American Society of Hematology 113 (4), 797-806, 2009 | 325 | 2009 |
| Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer J Bennicelli, JF Wright, A Komaromy, JB Jacobs, B Hauck, O Zelenaia, ... Molecular Therapy 16 (3), 458-465, 2008 | 316 | 2008 |
| Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver F Mingozzi, NC Hasbrouck, E Basner-Tschakarjan, SA Edmonson, DJ Hui, ... Blood, The Journal of the American Society of Hematology 110 (7), 2334-2341, 2007 | 276 | 2007 |
| Assessing the potential for AAV vector genotoxicity in a murine model H Li, N Malani, SR Hamilton, A Schlachterman, G Bussadori, ... Blood, The Journal of the American Society of Hematology 117 (12), 3311-3319, 2011 | 270 | 2011 |
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| Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation RW Herzog, JD Mount, VR Arruda, KA High, CD Lothrop Molecular Therapy 4 (3), 192-200, 2001 | 225 | 2001 |
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| Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy JD Finn, MC Ozelo, DE Sabatino, HWG Franck, EP Merricks, JM Crudele, ... Blood, The Journal of the American Society of Hematology 116 (26), 5842-5848, 2010 | 205 | 2010 |
