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Material Type: Artigo
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CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV VectorsWang, Dan ; Zhang, Feng ; Gao, GuangpingCell, 2020-04, Vol.181 (1), p.136-150 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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Material Type: Artigo
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In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic ModulationLiao, Hsin-Kai ; Hatanaka, Fumiyuki ; Araoka, Toshikazu ; Reddy, Pradeep ; Wu, Min-Zu ; Sui, Yinghui ; Yamauchi, Takayoshi ; Sakurai, Masahiro ; O’Keefe, David D. ; Núñez-Delicado, Estrella ; Guillen, Pedro ; Campistol, Josep M. ; Wu, Cheng-Jang ; Lu, Li-Fan ; Esteban, Concepcion Rodriguez ; Izpisua Belmonte, Juan CarlosCell, 2017-12, Vol.171 (7), p.1495-1507.e15 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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Material Type: Artigo
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Therapeutic in vivo delivery of gene editing agentsRaguram, Aditya ; Banskota, Samagya ; Liu, David R.Cell, 2022-07, Vol.185 (15), p.2806-2827 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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Material Type: Artigo
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Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophyLong, Chengzu ; Amoasii, Leonela ; Mireault, Alex A. ; McAnally, John R. ; Li, Hui ; Sanchez-Ortiz, Efrain ; Bhattacharyya, Samadrita ; Shelton, John M. ; Bassel-Duby, Rhonda ; Olson, Eric N.Science (American Association for the Advancement of Science), 2016-01, Vol.351 (6271), p.400-403 [Periódico revisado por pares]United States: American Association for the Advancement of ScienceTexto completo disponível |
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Material Type: Artigo
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In vivo gene editing in dystrophic mouse muscle and muscle stem cellsTabebordbar, Mohammadsharif ; Zhu, Kexian ; Cheng, Jason K. W. ; Chew, Wei Leong ; Widrick, Jeffrey J. ; Yan, Winston X. ; Maesner, Claire ; Wu, Elizabeth Y. ; Xiao, Ru ; Ran, F. Ann ; Le Cong ; Zhang, Feng ; Vandenberghe, Luk H. ; Church, George M. ; Wagers, Amy J.Science (American Association for the Advancement of Science), 2016-01, Vol.351 (6271), p.407-411 [Periódico revisado por pares]United States: American Association for the Advancement of ScienceTexto completo disponível |
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Material Type: Artigo
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Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich SyndromeAiuti, Alessandro ; Biasco, Luca ; Scaramuzza, Samantha ; Ferrua, Francesca ; Cicalese, Maria Pia ; Baricordi, Cristina ; Dionisio, Francesca ; Calabria, Andrea ; Giannelli, Stefania ; Castiello, Maria Carmina ; Bosticardo, Marita ; Evangelio, Costanza ; Assanelli, Andrea ; Casiraghi, Miriam ; Di Nunzio, Sara ; Callegaro, Luciano ; Benati, Claudia ; Rizzardi, Paolo ; Pellin, Danilo ; Di Serio, Clelia ; Schmidt, Manfred ; Von Kalle, Christof ; Gardner, Jason ; Mehta, Nalini ; Neduva, Victor ; Dow, David J. ; Galy, Anne ; Miniero, Roberto ; Finocchi, Andrea ; Metin, Ayse ; Banerjee, Pinaki P. ; Orange, Jordan S. ; Galimberti, Stefania ; Valsecchi, Maria Grazia ; Biffi, Alessandra ; Montini, Eugenio ; Villa, Anna ; Ciceri, Fabio ; Roncarolo, Maria Grazia ; Naldini, LuigiScience (American Association for the Advancement of Science), 2013-08, Vol.341 (6148), p.865-865 [Periódico revisado por pares]United States: American Association for the Advancement of ScienceTexto completo disponível |
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Material Type: Artigo
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Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic LeukodystrophyBiffi, Alessandra ; Montini, Eugenio ; Lorioli, Laura ; Cesani, Martina ; Fumagalli, Francesca ; Plati, Tiziana ; Baldoli, Cristina ; Martino, Sabata ; Calabria, Andrea ; Canale, Sabrina ; Benedicenti, Fabrizio ; Vallanti, Giuliana ; Biasco, Luca ; Leo, Simone ; Kabbara, Nabil ; Zanetti, Gianluigi ; Rizzo, William B. ; Mehta, Nalini A. L. ; Cicalese, Maria Pia ; Casiraghi, Miriam ; Boelens, Jaap J. ; Del Carro, Ubaldo ; Dow, David J. ; Schmidt, Manfred ; Assanelli, Andrea ; Neduva, Victor ; Di Serio, Clelia ; Stupka, Elia ; Gardner, Jason ; von Kalle, Christof ; Bordignon, Claudio ; Ciceri, Fabio ; Rovelli, Attilio ; Roncarolo, Maria Grazia ; Aiuti, Alessandro ; Sessa, Maria ; Naldini, LuigiScience (American Association for the Advancement of Science), 2013-08, Vol.341 (6148), p.864-864 [Periódico revisado por pares]United States: American Association for the Advancement of ScienceTexto completo disponível |
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Material Type: Artigo
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Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNALong, Chengzu ; McAnally, John R. ; Shelton, John M. ; Mireault, Alex A. ; Bassel-Duby, Rhonda ; Olson, Eric N.Science (American Association for the Advancement of Science), 2014-09, Vol.345 (6201), p.1184-1188 [Periódico revisado por pares]United States: American Association for the Advancement of ScienceTexto completo disponível |
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Material Type: Artigo
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Oncolytic Viro-Immunotherapy: An Emerging Option in the Treatment of GliomasZeng, Jiayi ; Li, Xiangxue ; Sander, Max ; Zhang, Haipeng ; Yan, Guangmei ; Lin, YuanFrontiers in immunology, 2021-10, Vol.12, p.721830-721830 [Periódico revisado por pares]Switzerland: Frontiers Media S.ATexto completo disponível |
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Material Type: Artigo
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Therapeutic targeting of preleukemia cells in a mouse model of NPM1 mutant acute myeloid leukemiaUckelmann, Hannah J ; Kim, Stephanie M ; Wong, Eric M ; Hatton, Charles ; Giovinazzo, Hugh ; Gadrey, Jayant Y ; Krivtsov, Andrei V ; Rücker, Frank G ; Döhner, Konstanze ; McGeehan, Gerard M ; Levine, Ross L ; Bullinger, Lars ; Vassiliou, George S ; Armstrong, Scott AScience (American Association for the Advancement of Science), 2020-01, Vol.367 (6477), p.586-590 [Periódico revisado por pares]United States: The American Association for the Advancement of ScienceTexto completo disponível |