Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome von Rai, Rajeev, Romito, Marianna, Rivers, Elizabeth, Turchiano, Giandomenico, Blattner, Georges, Vetharoy, Winston, Ladon, Dariusz, Andrieux, Geoffroy, Zhang, Fang, Zinicola, Marta, Leon-Rico, Diego, Santilli, Giorgia, Thrasher, Adrian J., Cavazza, Alessia

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Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells von Patsali, Petros, Turchiano, Giandomenico, Papasavva, Panayiota, Romito, Marianna, Loucari, Constantinos C, Stephanou, Coralea, Christou, Soteroulla, Sitarou, Maria, Mussolino, Claudio, Cornu, Tatjana I, Antoniou, Michael N, Lederer, Carsten W, Cathomen, Toni, Kleanthous, Marina

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Gene Editing and Genotoxicity: Targeting the Off-Targets von Blattner, Georges, Cavazza, Alessia, Thrasher, Adrian J., Turchiano, Giandomenico

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Correction: Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells von Turchiano, Giandomenico, Latella, Maria Carmela, Döring, Andreas Gogol, Cattoglio, Claudia, Mavilio, Fulvio, Izsvák, Zsuzsanna, Ivics, Zoltán, Recchia, Alessandra

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Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes von Latella, Maria Carmela, Cocchiarella, Fabienne, De Rosa, Laura, Turchiano, Giandomenico, Gonçalves, Manuel A.F.V., Larcher, Fernando, De Luca, Michele, Recchia, Alessandra

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Genomic analysis of Sleeping Beauty transposon integration in human somatic cells von Turchiano, Giandomenico, Latella, Maria Carmela, Gogol-Döring, Andreas, Cattoglio, Claudia, Mavilio, Fulvio, Izsvák, Zsuzsanna, Ivics, Zoltán, Recchia, Alessandra

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Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV von Vaidyanathan, Sriram, Kerschner, Jenny L., Paranjapye, Alekh, Sinha, Vrishti, Lin, Brian, Bedrosian, Tracy A., Thrasher, Adrian J., Turchiano, Giandomenico, Harris, Ann, Porteus, Matthew H.

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Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors von García, Marta, Bonafont, Jose, Martínez-Palacios, Jesús, Xu, Rudan, Turchiano, Giandomenico, Svensson, Stina, Thrasher, Adrian J., Larcher, Fernando, Del Rio, Marcela, Hernández-Alcoceba, Rubén, Garín, Marina I., Mencía, Ángeles, Murillas, Rodolfo

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Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq von Turchiano, Giandomenico, Andrieux, Geoffroy, Klermund, Julia, Blattner, Georges, Pennucci, Valentina, el Gaz, Melina, Monaco, Gianni, Poddar, Sushmita, Mussolino, Claudio, Cornu, Tatjana I., Boerries, Melanie, Cathomen, Toni

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Genome and Epigenome Editing to Treat Disorders of the Hematopoietic System von Mussolino, Claudio, Alzubi, Jamal, Pennucci, Valentina, Turchiano, Giandomenico, Cathomen, Toni

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Pigment epithelium-derived factor hinders photoreceptor cell death by reducing intracellular calcium in the degenerating retina von Comitato, Antonella, Subramanian, Preeti, Turchiano, Giandomenico, Montanari, Monica, Becerra, S. Patricia, Marigo, Valeria

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Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses von Frati, Giacomo, Brusson, Megane, Sartre, Gilles, Mlayah, Bochra, Felix, Tristan, Chalumeau, Anne, Antoniou, Panagiotis, Hardouin, Giulia, Concordet, Jean-Paul, Romano, Oriana, Turchiano, Giandomenico, Miccio, Annarita

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Dominant and recessive mutations in rhodopsin activate different cell death pathways von Comitato, Antonella, Di Salvo, Maria Teresa, Turchiano, Giandomenico, Montanari, Monica, Sakami, Sanae, Palczewski, Krzysztof, Marigo, Valeria

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Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells (vol 9, e112712, 2014) von Turchiano, Giandomenico, Latella, Maria Carmela, Doring, Andreas Gogol, Cattoglio, Claudia, Mavilio, Fulvio, Izsvak, Zsuzsanna, Ivics, Zoltan, Recchia, Alessandra

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Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia von Bahal, Sameer, Zinicola, Marta, Moula, Shefta E, Whittaker, Thomas E., Schejtman, Andrea, Naseem, Asma, Blanco, Elena, Vetharoy, Winston, Hu, Yi-Ting, Rai, Rajeev, Gomez-Castaneda, Eduardo, Cunha-Santos, Catarina, Burns, Siobhan O., Morris, Emma C., Booth, Claire, Turchiano, Giandomenico, Cavazza, Alessia, Thrasher, Adrian J., Santilli, Giorgia

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Failure to detect DNA-guided genome editing using Natronobacterium gregoryi Argonaute von Lee, Seung Hwan, Turchiano, Giandomenico, Ata, Hirotaka, Nowsheen, Somaira, Romito, Marianna, Lou, Zhenkun, Ryu, Seuk-Min, Ekker, Stephen C, Cathomen, Toni, Kim, Jin-Soo

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Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies von Frati, Giacomo, Antoniou, Panagiotis, Hardouin, Giulia, Mlaya, Bochra, Weber, Leslie, Felix, Tristan, Casini, Antonio, Rosello, Marion, Chalumeau, Anne, Amendola, Mario, Thrasher, Adrian J., Turchiano, Giandomenico, Del Bene, Filippo, El Nemer, Wassim, Cereseto, Anna, Concordet, Jean-Paul, Giovannangeli, Carine, Cavazzana, Marina, Miccio, Annarita

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A Highly Efficient and GMP Compliant Protocol to Manufacture CCR5 Edited Cells to Treat HIV Infection von Romito, Marianna, Meyer, Emily, Poddar, Sushmita, Heinz, Helena, Rositzka, Julia, Hildenbeutel, Markus, Gautron, Anne-Sophie, Busser, Brian W, Kok, Yik Lim, Turchiano, Giandomenico, Haas, Simone, Schäfer, Richard, Smith, Julianne, Metzner, Karin J, Mussolino, Claudio, Duchateau, Philippe, Cathomen, Toni, Cornu, Tatjana I

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125. Engineered Nucleases-Mediated In Situ Correction of a Genetic Defect By Homologous Recombination Into the Native Locus von Carmela Latella, Maria, Miselli, Francesca, Turchiano, Giandomenico, Mussolino, Claudio, Cudeiro, Javier, Larcher, Fernando, Recchia, Alessandra

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Progress and Harmonization of Gene Editing to Treat Human Diseases (Proceeding of COST Action CA21113 GenE-HumDi) von Cavazza, Alessia, Hendel, Ayal, Bak, Rasmus O, Rio, Paula, Güell, Marc, Lainšček, Duško, Arechavala-Gomeza, Virginia, Peng, Ling, Hapil, Fatma Zehra, Harvey, Joshua, Ortega, Francisco G, Gonzalez-Martinez, Coral, Lederer, Carsten W, Mikkelsen, Kasper, Gasiunas, Giedrius, Kalter, Nechama, Gonçalves, Manuel a F V, Petersen, Julie, Garanto, Alejandro, Montoliu, Lluis, Maresca, Marcello, Seemann, Stefan E, Gorodkin, Jan, Mazini, Loubna, Sanchez, Rosario, Rodriguez-Madoz, Juan R, Maldonado-Pérez, Noelia, Laura, Torella, Schmueck-Henneresse, Michael, Maccalli, Cristina, Grünewald, Julian, Carmona, Gloria, Kachamakova-Trojanowska, Neli, Miccio, Annarita, Martin, Francisco, Turchiano, Giandomenico, Cathomen, Toni, Luo, Yonglun, Tsai, Shengdar Q, Benabdellah, Karim

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