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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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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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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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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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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The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B von Soucie, J. Michael, Monahan, Paul E., Kulkarni, Roshni, Konkle, Barbara A., Mazepa, Marshall A.

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Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application von Monahan, Paul E, Lothrop, Clinton D, Sun, Junjiang, Hirsch, Matthew L, Kafri, Tal, Kantor, Boris, Sarkar, Rita, Tillson, D Michael, Elia, Joseph R, Samulski, R Jude

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Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose von Wu, Zhijian, Sun, Junjiang, Zhang, Taiping, Yin, Chaoying, Yin, Fang, Van Dyke, Terry, Samulski, Richard J, Monahan, Paul E

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Physical Functioning in Boys with Hemophilia in the U.S von Monahan, Paul E., MD, Baker, Judith R., MHSA, Riske, Brenda, MS, MBA, MPA, Soucie, J. Michael, PhD

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

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs von Sun, Junjiang, Shao, Wenwei, Chen, Xiaojing, Merricks, Elizabeth P., Wimsey, Lauren, Abajas, Yasmina L., Niemeyer, Glenn P., Lothrop, Clinton D., Monahan, Paul E., Samulski, R. Jude, Nichols, Timothy C., Li, Chengwen

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Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX von Sun, Junjiang, Hakobyan, Narine, Valentino, Leonard A., Feldman, Brian L., Samulski, R. Jude, Monahan, Paul E.

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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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Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua von Weber, Alfred, Engelmaier, Andrea, Voelkel, Dirk, Pachlinger, Robert, Scheiflinger, Friedrich, Monahan, Paul E., Rottensteiner, Hanspeter

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

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Factor IX: Insights from knock-out and genetically engineered mice 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., Negrier, Claude, Tarantino, Michael, Valentino, Leonard A., Mingozzi, Federico

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