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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High-performance genetically targetable optical neural silencing by light-driven proton pumps von Boyden, Edward S, Chow, Brian Y, Han, Xue, Dobry, Allison S, Qian, Xiaofeng, Chuong, Amy S, Li, Mingjie, Henninger, Michael A, Belfort, Gabriel M, Lin, Yingxi, Monahan, Patrick E

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An F876L mutation in androgen receptor confers genetic and phenotypic resistance to MDV3100 (enzalutamide) von Korpal, Manav, Korn, Joshua M, Gao, Xueliang, Rakiec, Daniel P, Ruddy, David A, Doshi, Shivang, Yuan, Jing, Kovats, Steve G, Kim, Sunkyu, Cooke, Vesselina G, Monahan, John E, Stegmeier, Frank, Roberts, Thomas M, Sellers, William R, Zhou, Wenlai, Zhu, Ping

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo von MCCARTY, D. M, FU, H, MONAHAN, P. E, TOULSON, C. E, NAIK, P, SAMULSKI, R. J

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Managing inventories in a two-echelon dual-channel supply chain von Kevin Chiang, Wei-yu, Monahan, George E.

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The effectiveness of passive sumps and static cowls in reducing radon levels in new build Irish dwellings von Monahan, E., Murphy, P., Long, S., Dowdall, A.

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FGFR genetic alterations predict for sensitivity to NVP-BGJ398, a selective pan-FGFR inhibitor von Guagnano, Vito, Kauffmann, Audrey, Wöhrle, Simon, Stamm, Christelle, Ito, Moriko, Barys, Louise, Pornon, Astrid, Yao, Yao, Li, Fang, Zhang, Yun, Chen, Zhi, Wilson, Christopher J, Bordas, Vincent, Le Douget, Mickaël, Gaither, L Alex, Borawski, Jason, Monahan, John E, Venkatesan, Kavitha, Brümmendorf, Thomas, Thomas, David M, Garcia-Echeverria, Carlos, Hofmann, Francesco, Sellers, William R, Graus-Porta, Diana

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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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Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia von Li, C, Narkbunnam, N, Samulski, R J, Asokan, A, Hu, G, Jacobson, L J, Manco-Johnson, M J, Monahan, P E

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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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IL‐6 receptor antagonist as adjunctive therapy with clotting factor replacement to protect against bleeding‐induced arthropathy in hemophilia von Narkbunnam, N., Sun, J., Hu, G., Lin, F.‐C., Bateman, T. A., Mihara, M., Monahan, P. E.

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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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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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Delayed Functional Expression of Neuronal Chemokine Receptors Following Focal Nerve Demyelination in the Rat: A Mechanism for the Development of Chronic Sensitization of Peripheral... von Bhangoo, Sonia, Ren, Dongjun, Miller, Richard J, Henry, Kenneth J, Lineswala, Jayana, Hamdouchi, Chafiq, Li, Baolin, Monahan, Patrick E, Chan, David M, Ripsch, Matthew S, White, Fletcher A

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Gene therapy in an era of emerging treatment options for hemophilia B von Monahan, P. 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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Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy von Keravala, A, Chavez, C L, Hu, G, Woodard, L E, Monahan, P E, Calos, M P

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Longitudinal alterations in bone morphometry, mechanical integrity and composition in Type-2 diabetes in a Zucker diabetic fatty (ZDF) rat von Monahan, Genna E., Schiavi-Tritz, Jessica, Britton, Marissa, Vaughan, Ted J.

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Joint bleeding in factor VIII deficient mice causes an acute loss of trabecular bone and calcification of joint soft tissues which is prevented with aggressive factor replacement von Lau, A. G., Sun, J., Hannah, W. B., Livingston, E. W., Heymann, D., Bateman, T. A., Monahan, P. E.

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