Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani, Amit C, Reiss, Ulreke M, Tuddenham, Edward G.D, Rosales, Cecilia, Chowdary, Pratima, McIntosh, Jenny, Della Peruta, Marco, Lheriteau, Elsa, Patel, Nishal, Raj, Deepak, Riddell, Anne, Pie, Jun, Rangarajan, Savita, Bevan, David, Recht, Michael, Shen, Yu-Min, Halka, Kathleen G, Basner-Tschakarjan, Etiena, Mingozzi, Federico, High, Katherine A, Allay, James, Kay, Mark A, Ng, Catherine Y.C, Zhou, Junfang, Cancio, Maria, Morton, Christopher L, Gray, John T, Srivastava, Deokumar, Nienhuis, Arthur W, Davidoff, Andrew M
Published in The New England journal of medicine (20.11.2014)
Published in The New England journal of medicine (20.11.2014)
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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
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.
Published in Blood (25.04.2013)
Published in Blood (25.04.2013)
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Genotoxicity of Retroviral Integration In Hematopoietic Cells
Nienhuis, Arthur W, Dunbar, Cynthia E, Sorrentino, Brian P
Published in Molecular therapy (01.06.2006)
Published in Molecular therapy (01.06.2006)
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Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial
Allay, James A, Sleep, Susan, Long, Scott, Tillman, David M, Clark, Rob, Carney, Gael, Fagone, Paolo, McIntosh, Jenny H, Nienhuis, Arthur W, Davidoff, Andrew M, Nathwani, Amit C, Gray, John T
Published in Human gene therapy (01.05.2011)
Published in Human gene therapy (01.05.2011)
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Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
Hematti, Peiman, Hong, Bum-Kee, Ferguson, Cole, Adler, Rima, Hanawa, Hideki, Sellers, Stephanie, Holt, Ingeborg E, Eckfeldt, Craig E, Sharma, Yugal, Schmidt, Manfred, von Kalle, Christof, Persons, Derek A, Billings, Eric M, Verfaillie, Catherine M, Nienhuis, Arthur W, Wolfsberg, Tyra G, Dunbar, Cynthia E, Calmels, Boris
Published in PLoS biology (01.12.2004)
Published in PLoS biology (01.12.2004)
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Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods
Fagone, Paolo, Wright, J Fraser, Nathwani, Amit C, Nienhuis, Arthur W, Davidoff, Andrew M, Gray, John T
Published in Human gene therapy. Part B. Methods (01.02.2012)
Published in Human gene therapy. Part B. Methods (01.02.2012)
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Gene Therapy for Hemophilia
Nienhuis, Arthur W., Nathwani, Amit C., Davidoff, Andrew M.
Published in Molecular therapy (03.05.2017)
Published in Molecular therapy (03.05.2017)
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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques
Nathwani, Amit C., Davidoff, Andrew M., Hanawa, Hideki, Hu, Yunyu, Hoffer, Fredric A., Nikanorov, Alexander, Slaughter, Clive, Ng, Catherine Y.C., Zhou, Junfang, Lozier, Jay N., Mandrell, Timothy D., Vanin, Elio F., Nienhuis, Arthur W.
Published in Blood (01.09.2002)
Published in Blood (01.09.2002)
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Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy
Wielgosz, Matthew M, Kim, Yoon-Sang, Carney, Gael G, Zhan, Jun, Reddivari, Muralidhar, Coop, Terry, Heath, Richard J, Brown, Scott A, Nienhuis, Arthur W
Published in Molecular therapy. Methods & clinical development (01.01.2015)
Published in Molecular therapy. Methods & clinical development (01.01.2015)
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Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
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
Published in The New England journal of medicine (22.12.2011)
Published in The New England journal of medicine (22.12.2011)
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Pathophysiology and Clinical Manifestations of the β-Thalassemias
Nienhuis, Arthur W, Nathan, David G
Published in Cold Spring Harbor perspectives in medicine (01.12.2012)
Published in Cold Spring Harbor perspectives in medicine (01.12.2012)
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Journal Article
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
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
Published in Molecular therapy (01.05.2011)
Published in Molecular therapy (01.05.2011)
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Journal Article
George Stamatoyannopoulos (1934–2018)
Dunbar, Cynthia E., Bodine, David M., Nienhuis, Arthur W.
Published in Molecular therapy (01.08.2018)
Published in Molecular therapy (01.08.2018)
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