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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Long-Term Effects of Hemophilia B Gene Therapy. Reply von Pipe, Steven W, Monahan, Paul E

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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A von George, Lindsey A, Monahan, Paul E, Eyster, M. Elaine, Sullivan, Spencer K, Ragni, Margaret V, Croteau, Stacy E, Rasko, John E.J, Recht, Michael, Samelson-Jones, Benjamin J, MacDougall, Amy, Jaworski, Kristen, Noble, Robert, Curran, Marla, Kuranda, Klaudia, Mingozzi, Federico, Chang, Tiffany, Reape, Kathleen Z, Anguela, Xavier M, High, Katherine A

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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector von Bowles, Dawn E, McPhee, Scott WJ, Li, Chengwen, Gray, Steven J, Samulski, Jade J, Camp, Angelique S, Li, Juan, Wang, Bing, Monahan, Paul E, Rabinowitz, Joseph E, Grieger, Joshua C, Govindasamy, Lakshmanan, Agbandje-McKenna, Mavis, Xiao, Xiao, Samulski, R Jude

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Men with severe hemophilia in the United States: birth cohort analysis of a large national database von Mazepa, Marshall A., Monahan, Paul E., Baker, Judith R., Riske, Brenda K., Soucie, J.Michael

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Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors von Monahan, Paul E

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Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia von Monahan, Paul E., Négrier, Claude, Tarantino, Michael, Valentino, Leonard A., Mingozzi, Federico

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FVIII at the crossroad of coagulation, bone and immune biology: Emerging evidence of biological activities beyond hemostasis von Cadé, Marie, Muñoz-Garcia, Javier, Babuty, Antoine, Fouassier, Marc, Heymann, Marie-Francoise, Monahan, Paul E., Heymann, Dominique

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Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice von Cooley, Brian, Funkhouser, William, Monroe, Dougald, Ezzell, Ashley, Mann, David M., Lin, Feng-Chang, Monahan, Paul E., Stafford, Darrel W.

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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-assoc... von Monahan, Paul E, Sun, Junjiang, Gui, Tong, Hu, Genlin, Hannah, William B, Wichlan, David G, Wu, Zhijian, Grieger, Joshua C, Li, Chengwen, Suwanmanee, Thipparat, Stafford, Darrel W, Booth, Carmen J, Samulski, Jade J, Kafri, Tal, McPhee, Scott W J, Samulski, R Jude

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Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half‐life factor IX therapies for severe or moderately severe haemophilia B von Klamroth, Robert, Bonner, Ashley, Gomez, Keith, Monahan, Paul E., Szafranski, Kirk, Zhang, Xiang, Walsh, Sarah, Wang, Di, Yan, Songkai

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Exploring the Potential Feasibility of Intra-Articular Adeno-Associated Virus-Mediated Gene Therapy for Hemophilia Arthropathy von Zhang, Feixu, Yan, Xiaobo, Li, Min, Hua, Baolai, Xiao, Xiao, Monahan, Paul E, Sun, Junjiang

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Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B von Schmidt, Manfred, Foster, Graham R., Coppens, Michiel, Thomsen, Hauke, Dolmetsch, Ricardo, Heijink, Liesbeth, Monahan, Paul E., Pipe, Steven W.

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Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles von CHENGWEN LI, DIPRIMIO, Nina, BOWLES, Dawn E, HIRSCH, Matthew L, MONAHAN, Paul E, ASOKAN, Aravind, RABINOWITZ, Joseph, AGBANDJE-MCKENNA, Mavis, JUDE SAMULSKI, R

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Discussing investigational AAV gene therapy with hemophilia patients: A guide von Sidonio, Robert F., Pipe, Steven W., Callaghan, Michael U., Valentino, Leonard A., Monahan, Paul E., Croteau, Stacy E.

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Abnormal joint and bone wound healing in hemophilia mice is improved by extending factor IX activity after hemarthrosis von Sun, Junjiang, Hua, Baolai, Livingston, Eric W., Taves, Sarah, Johansen, Peter B., Hoffman, Maureane, Ezban, Mirella, Monroe, Dougald M., Bateman, Ted A., Monahan, Paul E.

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Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B von O’Connell, Niamh, van der Valk, Paul, Le Quellec, Sandra, Gomez, Esteban, Monahan, Paul E., Crary, Shelley E., Coppens, Michiel, Lemons, Richard, Castaman, Giancarlo, Klamroth, Robert, Symington, Emily, Quon, Doris V., Kampmann, Peter

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Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene thera... von Itzler, Robbin, Buckner, Tyler W, Leebeek, Frank W G, Miller, Joel, Recht, Michael, Drelich, Douglass, Monahan, Paul E, Pipe, Steven W

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Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX von Ramaswamy, Suvasini, Tonnu, Nina, Menon, Tushar, Lewis, Benjamin M., Green, Kevin T., Wampler, Derek, Monahan, Paul E., Verma, Inder M.

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Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection von Kantor, Boris, Ma, Hong, Webster-Cyriaque, Jennifer, Monahan, Paul E, Kafri, Tal

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