Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Hinderer, Christian, Katz, Nathan, Buza, Elizabeth L, Dyer, Cecilia, Goode, Tamara, Bell, Peter, Richman, Laura K, Wilson, James M
Published in Human gene therapy (01.03.2018)
Published in Human gene therapy (01.03.2018)
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Journal Article
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
Hordeaux, Juliette, Buza, Elizabeth L, Dyer, Cecilia, Goode, Tamara, Mitchell, Thomas W, Richman, Laura, Denton, Nathan, Hinderer, Christian, Katz, Nathan, Schmid, Ralf, Miller, Rod, Choudhury, Gourav R, Horiuchi, Makoto, Nambiar, Kalyani, Yan, Hanying, Li, Mingyao, Wilson, James M
Published in Human gene therapy (01.08.2020)
Published in Human gene therapy (01.08.2020)
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The First Approved Gene Therapy Product for Cancer Ad-p53 (Gendicine): 12 Years in the Clinic
Zhang, Wei-Wei, Li, Longjiang, Li, Dinggang, Liu, Jiliang, Li, Xiuqin, Li, Wei, Xu, Xiaolong, Zhang, Michael J, Chandler, Lois A, Lin, Hong, Hu, Aiguo, Xu, Wei, Lam, Dominic Man-Kit
Published in Human gene therapy (01.02.2018)
Published in Human gene therapy (01.02.2018)
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Effective Targeting of Multiple B-Cell Maturation Antigen-Expressing Hematological Malignances by Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T Cells
Friedman, Kevin M, Garrett, Tracy E, Evans, John W, Horton, Holly M, Latimer, Howard J, Seidel, Stacie L, Horvath, Christopher J, Morgan, Richard A
Published in Human gene therapy (01.05.2018)
Published in Human gene therapy (01.05.2018)
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Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses
Wang, Dan, Mou, Haiwei, Li, Shaoyong, Li, Yingxiang, Hough, Soren, Tran, Karen, Li, Jia, Yin, Hao, Anderson, Daniel G, Sontheimer, Erik J, Weng, Zhiping, Gao, Guangping, Xue, Wen
Published in Human gene therapy (01.07.2015)
Published in Human gene therapy (01.07.2015)
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Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Hocquemiller, Michaël, Giersch, Laura, Audrain, Mickael, Parker, Samantha, Cartier, Nathalie
Published in Human gene therapy (01.07.2016)
Published in Human gene therapy (01.07.2016)
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Nucleofection with Plasmid DNA for CRISPR/Cas9-Mediated Inactivation of Programmed Cell Death Protein 1 in CD133-Specific CAR T Cells
Hu, Bian, Zou, Yan, Zhang, Linlin, Tang, Jiaxing, Niedermann, Gabriele, Firat, Elke, Huang, Xingxu, Zhu, Xuekai
Published in Human gene therapy (01.04.2019)
Published in Human gene therapy (01.04.2019)
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Public Acceptability of Gene Therapy and Gene Editing for Human Use: A Systematic Review
Delhove, Juliette, Osenk, Ivana, Prichard, Ivanka, Donnelley, Martin
Published in Human gene therapy (01.01.2020)
Published in Human gene therapy (01.01.2020)
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Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study
Campochiaro, Peter A, Lauer, Andreas K, Sohn, Elliott H, Mir, Tahreem A, Naylor, Stuart, Anderton, Matthew C, Kelleher, Michelle, Harrop, Richard, Ellis, Scott, Mitrophanous, Kyriacos A
Published in Human gene therapy (01.01.2017)
Published in Human gene therapy (01.01.2017)
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Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A
Klamroth, Robert, Hayes, Gregory, Andreeva, Tatiana, Gregg, Keith, Suzuki, Takashi, Mitha, Ismail Haroon, Hardesty, Brandon, Shima, Midori, Pollock, Toni, Slev, Patricia, Oldenburg, Johannes, Ozelo, Margareth C, Stieltjes, Natalie, Castet, Sabine-Marie, Mahlangu, Johnny, Peyvandi, Flora, Kazmi, Rashid, Schved, Jean-François, Leavitt, Andrew D, Callaghan, Michael, Pan-Petesch, Brigitte, Quon, Doris V, Andrews, Jayson, Trinh, Alex, Li, Mingjin, Wong, Wing Yen
Published in Human gene therapy (01.04.2022)
Published in Human gene therapy (01.04.2022)
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Journal Article
Re: "Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy" by Wilson and Flotte
Shieh, Perry B, Bönnemann, Carsten G, Müller-Felber, Wolfgang, Blaschek, Astrid, Dowling, James J, Kuntz, Nancy L, Seferian, Andreea M
Published in Human gene therapy (01.08.2020)
Published in Human gene therapy (01.08.2020)
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Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity
Tran, Ngoc Tam, Lecomte, Emilie, Saleun, Sylvie, Namkung, Suk, Robin, Cécile, Weber, Kristina, Devine, Eric, Blouin, Veronique, Adjali, Oumeya, Ayuso, Eduard, Gao, Guangping, Penaud-Budloo, Magalie, Tai, Phillip W L
Published in Human gene therapy (01.04.2022)
Published in Human gene therapy (01.04.2022)
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Postmortem Analysis in a Clinical Trial of AAV2-NGF Gene Therapy for Alzheimer's Disease Identifies a Need for Improved Vector Delivery
Castle, Michael J, Baltanás, Fernando C, Kovacs, Imre, Nagahara, Alan H, Barba, David, Tuszynski, Mark H
Published in Human gene therapy (01.04.2020)
Published in Human gene therapy (01.04.2020)
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