Gene Therapy von High, Katherine A, Roncarolo, Maria G

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Gene therapy von Brody, Herb

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Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products von Aiuti, Alessandro, Roncarolo, Maria Grazia, Naldini, Luigi

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DNAzyme‐Loaded Metal–Organic Frameworks (MOFs) for Self‐Sufficient Gene Therapy von Wang, Huimin, Chen, Yuqi, Wang, Hong, Liu, Xiaoqing, Zhou, Xiang, Wang, Fuan

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Gene therapy comes of age von Dunbar, Cynthia E, High, Katherine A, Joung, J Keith, Kohn, Donald B, Ozawa, Keiya, Sadelain, Michel

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CRISPR/Cas gene therapy von Zhang, Baohong

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Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene von Negre, Olivier, Eggimann, Anne-Virginie, Beuzard, Yves, Ribeil, Jean-Antoine, Bourget, Philippe, Borwornpinyo, Suparerk, Hongeng, Suradej, Hacein-Bey, Salima, Cavazzana, Marina, Leboulch, Philippe, Payen, Emmanuel

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History of gene therapy von Wirth, Thomas, Parker, Nigel, Ylä-Herttuala, Seppo

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Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial von Fischer, M. Dominik, Michalakis, Stylianos, Wilhelm, Barbara, Zobor, Ditta, Muehlfriedel, Regine, Kohl, Susanne, Weisschuh, Nicole, Ochakovski, G. Alex, Klein, Reinhild, Schoen, Christian, Sothilingam, Vithiyanjali, Garcia-Garrido, Marina, Kuehlewein, Laura, Kahle, Nadine, Werner, Annette, Dauletbekov, Daniyar, Paquet-Durand, François, Tsang, Stephen, Martus, Peter, Peters, Tobias, Seeliger, Mathias, Bartz-Schmidt, Karl Ulrich, Ueffing, Marius, Zrenner, Eberhart, Biel, Martin, Wissinger, Bernd

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Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results von Feuer, William J, Schiffman, Joyce C, Davis, Janet L, Porciatti, Vittorio, Gonzalez, Phillip, Koilkonda, Rajeshwari D, Yuan, Huijun, Lalwani, Anil, Lam, Byron L, Guy, John

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Gene therapy clinical trials worldwide to 2017: An update von Ginn, Samantha L., Amaya, Anais K., Alexander, Ian E., Edelstein, Michael, Abedi, Mohammad R.

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Ex vivo gene therapy using human bone marrow cells overexpressing BMP-2: “Next-day” gene therapy versus standard “two-step” approach von Bougioukli, Sofia, Alluri, Ram, Pannell, William, Sugiyama, Osamu, Vega, Andrew, Tang, Amy, Skorka, Tautis, Park, Sang Hyun, Oakes, Daniel, Lieberman, Jay R.

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Human Cardiac Gene Therapy von Ishikawa, Kiyotake, Weber, Thomas, Hajjar, Roger J

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Gene therapy for hemophilia von Nathwani, Amit C

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Gene therapy in cancer von Cesur-Ergün, Büşra, Demir-Dora, Devrim

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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A von Ozelo, Margareth C, Mahlangu, Johnny, Pasi, K. John, Giermasz, Adam, Leavitt, Andrew D, Laffan, Michael, Symington, Emily, Quon, Doris V, Wang, Jiaan-Der, Peerlinck, Kathelijne, Pipe, Steven W, Madan, Bella, Key, Nigel S, Pierce, Glenn F, O’Mahony, Brian, Kaczmarek, Radoslaw, Henshaw, Joshua, Lawal, Adebayo, Jayaram, Kala, Huang, Mei, Yang, Xinqun, Wong, Wing Y, Kim, Benjamin

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Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis von Bainbridge, James W.B, Mehat, Manjit S, Sundaram, Venki, Robbie, Scott J, Barker, Susie E, Ripamonti, Caterina, Georgiadis, Anastasios, Mowat, Freya M, Beattie, Stuart G, Gardner, Peter J, Feathers, Kecia L, Luong, Vy A, Yzer, Suzanne, Balaggan, Kamaljit, Viswanathan, Ananth, de Ravel, Thomy J.L, Casteels, Ingele, Holder, Graham E, Tyler, Nick, Fitzke, Fred W, Weleber, Richard G, Nardini, Marko, Moore, Anthony T, Thompson, Debra A, Petersen-Jones, Simon M, Michaelides, Michel, van den Born, L. Ingeborgh, Stockman, Andrew, Smith, Alexander J, Rubin, Gary, Ali, Robin R

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Immune Responses to Viral Gene Therapy Vectors von Shirley, Jamie L., de Jong, Ype P., Terhorst, Cox, Herzog, Roland W.

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Gene Therapy for Neurodegenerative Diseases von Sudhakar, Vivek, Richardson, R. Mark

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Gene therapy returns to centre stage von Naldini, Luigi

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