BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression von Konkle, Barbara A., Walsh, Christopher E., Escobar, Miguel A., Josephson, Neil C., Young, Guy, von Drygalski, Annette, McPhee, Scott W.J., Samulski, R. Jude, Bilic, Ivan, de la Rosa, Maurus, Reipert, Birgit M., Rottensteiner, Hanspeter, Scheiflinger, Friedrich, Chapin, John C., Ewenstein, Bruce, Monahan, Paul E.

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Impact of inhibitors on hemophilia a mortality in the United States von Walsh, Christopher E., Soucie, J. Michael, Miller, Connie H.

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Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial von Collins, Peter W., Young, Guy, Knobe, Karin, Karim, Faraizah Abdul, Angchaisuksiri, Pantep, Banner, Claus, Gürsel, Türkiz, Mahlangu, Johnny, Matsushita, Tadashi, Mauser-Bunschoten, Eveline P., Oldenburg, Johannes, Walsh, Christopher E., Negrier, Claude

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Acquired hemophilia: a rare but life-threatening potential cause of bleeding in the intensive care unit von Shander, Aryeh, Walsh, Christopher E., Cromwell, Caroline

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Several Log Increase in Therapeutic Transgene Delivery by Distinct Adeno-Associated Viral Serotype Vectors von Chao, Hengjun, Liu, Yuanbo, Rabinowitz, Joseph, Li, Chengwen, Samulski, Richard Jude, Walsh, Christopher E.

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The burden of inhibitors in haemophilia patients von Walsh, Christopher E., Jiménez-Yuste, Victor, Auerswald, Guenter, Grancha, Salvador

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Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing von Chao, Hengjun, Mansfield, S Gary, Bartel, Robert C, Hiriyanna, Suja, Mitchell, Lloyd G, Garcia-Blanco, Mariano A, Walsh, Christopher E

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A systematic review of adeno‐associated virus gene therapy clinical trials for HIV – A potential solution for patients with haemophilia and HIV? von Levy‐Mendelovich, Sarina, Walsh, Christopher E., Schapiro, Jonathan M., Soffer, Shelly, Klang, Eyal, Kenet, Gili

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Targeted integration of a rAAV vector into the AAVS1 region von Ward, Peter, Walsh, Christopher E

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Sustained and Complete Phenotype Correction of Hemophilia B Mice Following Intramuscular Injection of AAV1 Serotype Vectors von Chao, Hengjun, Monahan, Paul E., Liu, Yuanbo, Samulski, R.Jude, Walsh, Christopher E.

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Lentivirus Vector Purification Using Anion Exchange HPLC Leads to Improved Gene Transfer von Yamada, Kaoru, McCarty, Douglas M, Madden, Victoria J, Walsh, Christopher E

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Expression of Human Factor VIII by Splicing between Dimerized AAV Vectors von Chao, Hengjun, Sun, Liangwu, Bruce, Andrew, Xiao, Xiao, Walsh, Christopher E.

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Optimizing liver health before and after gene therapy for hemophilia A von Ragni, Margaret V., Mead, Henry, de Jong, Ype P., Kaczmarek, Radoslaw, Leavitt, Andrew D., Long, Brian, Nugent, Diane J., Sabatino, Denise E., Fong, Sylvia, von Drygalski, Annette, Walsh, Christopher E., Luxon, Bruce A.

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Safe and Sustained Overexpression of Functional Apolipoprotein A-I/High-density Lipoprotein in Apolipoprotein A-I–null Mice by Muscular Adeno-associated Viral Serotype 8 Vector Gen... von Cimmino, Giovanni, Chen, Wei, Speidl, Walter S, Giannarelli, Chiara, Ibanez, Borja, Fuster, Valentin, Hajjar, Roger, Walsh, Christopher E, Badimon, Juan J

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Hemophilia clinical gene therapy: brief review von Walsh, Christopher E, Batt, Katherine M

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Factor IX ectopically expressed in platelets can be stored in α-granules and corrects the phenotype of hemophilia B mice von Zhang, Guowei, Shi, Qizhen, Fahs, Scot A., Kuether, Erin L., Walsh, Christopher E., Montgomery, Robert R.

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Spliceosome-mediated RNA trans-splicing von Yang, Yanping, Walsh, Christopher E

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Phenotypes of allo- and autoimmune antibody responses to FVIII characterized by surface plasmon resonance von Lewis, Kenneth B, Hughes, Richard J, Epstein, Melinda S, Josephson, Neil C, Kempton, Christine L, Kessler, Craig M, Key, Nigel S, Howard, Tom E, Kruse-Jarres, Rebecca, Lusher, Jeanne M, Walsh, Christopher E, Watts, Raymond G, Ettinger, Ruth A, Pratt, Kathleen P

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Efficacy of EHL N9-GP for on-demand treatment of bleeding episodes in hemophilia B: analysis of pivotal trial data von Escobar, Miguel A, Walsh, Christopher E, Cooper, David L, Young, Guy

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Chimeric AAV Cap sequences alter gene transduction von Ward, Peter, Walsh, Christopher E

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