Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B von Nathwani, Amit C, Tuddenham, Edward G.D, Rangarajan, Savita, Rosales, Cecilia, McIntosh, Jenny, Linch, David C, Chowdary, Pratima, Riddell, Anne, Pie, Arnulfo Jaquilmac, Harrington, Chris, O'Beirne, James, Smith, Keith, Pasi, John, Glader, Bertil, Rustagi, Pradip, Ng, Catherine Y.C, Kay, Mark A, Zhou, Junfang, Spence, Yunyu, Morton, Christopher L, Allay, James, Coleman, John, Sleep, Susan, Cunningham, John M, Srivastava, Deokumar, Basner-Tschakarjan, Etiena, Mingozzi, Federico, High, Katherine A, Gray, John T, Reiss, Ulrike M, Nienhuis, Arthur W, Davidoff, Andrew M

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Codon optimization of human factor VIII cDNAs leads to high-level expression von Ward, Natalie J., Buckley, Suzanne M.K., Waddington, Simon N., VandenDriessche, Thierry, Chuah, Marinee K.L., Nathwani, Amit C., McIntosh, Jenny, Tuddenham, Edward G.D., Kinnon, Christine, Thrasher, Adrian J., McVey, John H.

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Mutations in VKORC1 cause warfarin resistance and multiple coagulation factor deficiency type 2 von Oldenburg, Johannes, Rost, Simone, Fregin, Andreas, Ivaskevicius, Vytautas, Conzelmann, Ernst, Hörtnagel, Konstanze, Pelz, Hans-Joachim, Lappegard, Knut, Seifried, Erhard, Scharrer, Inge, Tuddenham, Edward G. D, Müller, Clemens R, Strom, Tim M

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Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman prima... von Nathwani, Amit C., Gray, John T., McIntosh, Jenny, Ng, Catherine Y.C., Zhou, Junfang, Spence, Yunyu, Cochrane, Melanie, Gray, Elaine, Tuddenham, Edward G.D., Davidoff, Andrew M.

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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant von McIntosh, Jenny, Lenting, Peter J., Rosales, Cecilia, Lee, Doyoung, Rabbanian, Samira, Raj, Deepak, Patel, Nishil, Tuddenham, Edward G.D., Christophe, Olivier D., McVey, John H., Waddington, Simon, Nienhuis, Arthur W., Gray, John T., Fagone, Paolo, Mingozzi, Federico, Zhou, Shang-Zhen, High, Katherine A., Cancio, Maria, Ng, Catherine Y.C., Zhou, Junfang, Morton, Christopher L., Davidoff, Andrew M., Nathwani, Amit C.

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Genotype-phenotype correlation in combined deficiency of factor V and factor VIII von Zhang, Bin, Spreafico, Marta, Zheng, Chunlei, Yang, Angela, Platzer, Petra, Callaghan, Michael U., Avci, Zekai, Ozbek, Namik, Mahlangu, Johnny, Haw, Tabitha, Kaufman, Randal J., Marchant, Kandice, Tuddenham, Edward G.D., Seligsohn, Uri, Peyvandi, Flora, Ginsburg, David

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Solution structure of the major factor VIII binding region on von Willebrand factor von Shiltagh, Nuha, Kirkpatrick, John, Cabrita, Lisa D., McKinnon, Tom A.J., Thalassinos, Konstantinos, Tuddenham, Edward G.D., Hansen, D. Flemming

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Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman... von Nathwani, Amit C., Gray, John T., Ng, Catherine Y.C., Zhou, Junfang, Spence, Yunyu, Waddington, Simon N., Tuddenham, Edward G.D., Kemball-Cook, Geoffrey, McIntosh, Jenny, Boon-Spijker, Mariette, Mertens, Koen, Davidoff, Andrew M.

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Live birth following the first mutation specific pre-implantation genetic diagnosis for haemophilia A von Michaelides, Katerina, Tuddenham, Edward G. D., Turner, Cathy, Lavender, Ben, Lavery, Stuart A.

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The complex, confusing and poorly understood immune responses to AAV-mediated gene transfer in haemophilia-Is more or less immunosuppression required? von Tuddenham, Edward G D, Foster, Graham R

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AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage von Binny, Christopher, McIntosh, Jenny, Della Peruta, Marco, Kymalainen, Hanna, Tuddenham, Edward G.D., Buckley, Suzanne M.K., Waddington, Simon N., McVey, John H., Spence, Yunyu, Morton, Christopher L., Thrasher, Adrian J., Gray, John T., Castellino, Francis J., Tarantal, Alice F., Davidoff, Andrew M., Nathwani, Amit C.

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Factor VIII: the protein, cloning its gene, synthetic factor and now – 35 years later – gene therapy; what happened in between? von Ling, Gavin, Tuddenham, Edward G. D.

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Recent advances in developing specific therapies for haemophilia von Ling, Gavin, Nathwani, Amit C., Tuddenham, Edward G. D.

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Advances in Gene Therapy for Hemophilia von Nathwani, Amit C, Davidoff, Andrew M, Tuddenham, Edward G D

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Haemophilia, the journey in search of a cure. 1960–2020 von Nathwani, Amit C., Tuddenham, Edward G. D.

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Mathematical models of coagulation—are we there yet? von Owen, Matt J., Wright, Joy R., Tuddenham, Edward G.D., King, John R., Goodall, Alison H., Dunster, Joanne L.

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Combined deficiency of factor V and factor VIII is due to mutations in either LMAN1 or MCFD2 von Zhang, Bin, McGee, Beth, Yamaoka, Jennifer S., Guglielmone, Hugo, Downes, Katharine A., Minoldo, Salvador, Jarchum, Gustavo, Peyvandi, Flora, de Bosch, Norma B., Ruiz-Saez, Arlette, Chatelain, Bernard, Olpinski, Marian, Bockenstedt, Paula, Sperl, Wolfgang, Kaufman, Randal J., Nichols, William C., Tuddenham, Edward G.D., Ginsburg, David

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A novel missense mutation in ABCA1 results in altered protein trafficking and reduced phosphatidylserine translocation in a patient with Scott syndrome von Albrecht, Christiane, McVey, John H., Elliott, James I., Sardini, Alessandro, Kasza, Ildiko, Mumford, Andrew D., Naoumova, Rossi P., Tuddenham, Edward G.D., Szabo, Katalin, Higgins, Christopher F.

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A common ancestral glycoprotein ( GP) 9 1828A>G (Asn45Ser) gene mutation occurring in European families from Australia and Northern Europe with Bernard-Soulier syndrome (BSS) von Helena Liang, Hai Po, Morel-Kopp, Marie-Christine, Clemetson, Jeannine M., Clemetson, Kenneth J., Kekomaki, Riitta, Kroll, Hartmut, Michaelides, Katerina, Tuddenham, Edward G. D., Vanhoorelbeke, Karen, Ward, Christopher M.

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The Hemophilias — From Royal Genes to Gene Therapy von Mannucci, Pier M, Tuddenham, Edward G.D

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