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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TNNT1 nemaline myopathy: natural history and therapeutic frontier von Fox, Michael D, Carson, Vincent J, Feng, Han-Zhong, Lawlor, Michael W, Gray, John T, Brigatti, Karlla W, Jin, J -P, Strauss, Kevin A

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Design and Construction of Functional AAV Vectors von Gray, John T., Zolotukhin, Serge

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Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial von ALLAY, James A, SLEEP, Susan, NATHWANI, Amit C, GRAY, John T, LONG, Scott, TILLMAN, David M, CLARK, Rob, CARNEY, Gael, FAGONE, Paolo, MCINTOSH, Jenny H, NIENHUIS, Arthur W, DAVIDOFF, Andrew 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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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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Nfix is a novel regulator of murine hematopoietic stem and progenitor cell survival von Holmfeldt, Per, Pardieck, Jennifer, Saulsberry, Anjelica C., Nandakumar, Satish Kumar, Finkelstein, David, Gray, John T., Persons, Derek A., McKinney-Freeman, Shannon

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Induction of Neuron-Specific Degradation of Coenzyme A Models Pantothenate Kinase-Associated Neurodegeneration by Reducing Motor Coordination in Mice von Shumar, Stephanie A, Fagone, Paolo, Alfonso-Pecchio, Adolfo, Gray, John T, Rehg, Jerold E, Jackowski, Suzanne, Leonardi, Roberta

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Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection von Throm, Robert E., Ouma, Annastasia A., Zhou, Sheng, Chandrasekaran, Anantharaman, Lockey, Timothy, Greene, Michael, De Ravin, Suk See, Moayeri, Morvarid, Malech, Harry L., Sorrentino, Brian P., Gray, John T.

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Exogenous control of mammalian gene expression through modulation of RNA self-cleavage von Mulligan, Richard C, Yen, Laising, Svendsen, Jennifer, Lee, Jeng-Shin, Gray, John T, Magnier, Maxime, Baba, Takashi, D'Amato, Robert J

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Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector von Zhou, Sheng, Ma, Zhijun, Lu, Taihe, Janke, Laura, Gray, John T, Sorrentino, Brian P

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Soluble receptor-mediated selective inhibition of VEGFR and PDGFRβ signaling during physiologic and tumor angiogenesis von Kuhnert, Frank, Tam, Betty Y.Y, Sennino, Barbara, Gray, John T, Yuan, Jenny, Jocson, Angeline, Nayak, Nihar R, Mulligan, Richard C, McDonald, Donald M, Kuo, Calvin J

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Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps von Brimble, Mark A., Cheng, Pei-Hsin, Winston, Stephen M., Reeves, Isaiah L., Souquette, Aisha, Spence, Yunyu, Zhou, Junfang, Wang, Yong-Dong, Morton, Christopher L., Valentine, Marcus, Thomas, Paul G., Nathwani, Amit C., Gray, John T., Davidoff, Andrew M.

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An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation von Ryu, Byoung Y., Evans-Galea, Marguerite V., Gray, John T., Bodine, David M., Persons, Derek A., Nienhuis, Arthur W.

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Efficiency of transduction of highly purified murine hematopoietic stem cells by lentiviral and oncoretroviral vectors under conditions of minimal in vitro manipulation von Mostoslavsky, Gustavo, Kotton, Darrell N, Fabian, Attila J, Gray, John T, Lee, Jeng-Shin, Mulligan, Richard C

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Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods von FAGONE, Paolo, FRASER WRIGHT, J, NATHWANI, Amit C, NIENHUIS, Arthur W, DAVIDOFF, Andrew M, GRAY, John T

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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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Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome von Greig, Jenny A., Nordin, Jayme M.L., Draper, Christine, McMenamin, Deirdre, Chroscinski, Edward A., Bell, Peter, Gray, John T., Richman, Laura K., Wilson, James M.

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Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins von Nathwani, Amit C, Rosales, Cecilia, McIntosh, Jenny, Rastegarlari, Ghasem, Nathwani, Devhrut, Raj, Deepak, Nawathe, Sushmita, Waddington, Simon N, Bronson, Roderick, Jackson, Scott, Donahue, Robert E, High, Katherine A, Mingozzi, Federico, Ng, Catherine YC, Zhou, Junfang, Spence, Yunyu, McCarville, M Beth, Valentine, Marc, Allay, James, Coleman, John, Sleep, Susan, Gray, John T, Nienhuis, Arthur W, Davidoff, Andrew M

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Adeno-Associated Virus Vector-Mediated Systemic Delivery of IFN-β Combined with Low-Dose Cyclophosphamide Affects Tumor Regression in Murine Neuroblastoma Models von STRECK, Christian J, DICKSON, Paxton V, NG, Catherine Y. C, JUNFANG ZHOU, GRAY, John T, NATHWANI, Amit C, DAVIDOFF, Andrew M

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