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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Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis von MCCARTY, D. M, MONAHAN, P. E, SAMULSKI, R. J

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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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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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AAV vectors : is clinical success on the horizon? von MONAHAN, P. E, SAMULSKI, R. J

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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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Gene therapy in an era of emerging treatment options for hemophilia B von Monahan, P. E.

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Off-label use of recombinant factor VIIa in patients following bone marrow transplantation von BLATT, J, GOLD, S. H, WILEY, J. M, MONAHAN, P. E, COOPER, H. C, HARVEY, D

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Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia von MONAHAN, P. E, SAMULSKI, R. J, TAZELAAR, J, XIAO, X, NICHOLS, T. C, BELLINGER, D. A, READ, M. S, WALSH, C. E

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Prevalent inhibitors in haemophilia B subjects enrolled in the Universal Data Collection database von Puetz, J., Soucie, J. M., Kempton, C. L., Monahan, P. E.

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The expanding menagerie: animal models of hemophilia A von MONAHAN, P. E.

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Inhibitor recurrence after immune tolerance induction: a multicenter retrospective cohort study von Antun, A., Monahan, P. E., Manco‐Johnson, M. J., Callaghan, M. U., Kanin, M., Knoll, C., Carpenter, S. L., Davis, J. A., Guerrera, M. F., Kruse‐Jarres, R., Ragni, M. V., Witmer, C., McCracken, C. E., Kempton, C. L.

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Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant mice von ZHANG, T.-P, JIN, D.-Y, WARDROP, R. M, GUI, T, MAILE, R, FRELINGER, J. A, STAFFORD, D. W, MONAHAN, P. E

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Persistent expression of canine factor IX in hemophilia B canines von CHAO, H, SAMULSKI, R. J, BELLINGER, D. A, MONAHAN, P. E, NICHOLS, T. C, WALSH, C. E

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Recombinant porcine factor VIII for high‐risk surgery in paediatric congenital haemophilia A with high‐titre inhibitor von Croteau, S. E., Abajas, Y. L., Wolberg, A. S., Nielsen, B. I., Marx, G. R., Baird, C. W., Neufeld, E. J., Monahan, P. E.

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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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Abnormal hemostasis in a knock‐in mouse carrying a variant of factor IX with impaired binding to collagen type IV von GUI, T., REHEMAN, A., NI, H., GROSS, P. L., YIN, F., MONROE, D., MONAHAN, P. E., STAFFORD, D. W.

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Familial deficiency of vitamin K-dependent clotting factors von WESTON, B. W., MONAHAN, P. E.

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Safety and efficacy of investigator-prescribed BeneFIX® prophylaxis in children less than 6 years of age with severe haemophilia B von MONAHAN, P. E., LIESNER, R., SULLIVAN, S. T., RAMIREZ, M. E., KELLY, P., ROTH, D. A.

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Consequences of intra-articular bleeding in haemophilia: science to clinical practice and beyond von FORSYTH, A. L., RIVARD, G.-É., VALENTINO, L. A., ZOURIKIAN, N., HOFFMAN, M., MONAHAN, P. E., VAN MEEGEREN, M. E. R., FORRIOL, F.

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