Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases von Poletti, Valentina, Mavilio, Fulvio

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Nuclear architecture dictates HIV-1 integration site selection von Marini, Bruna, Kertesz-Farkas, Attila, Ali, Hashim, Lucic, Bojana, Lisek, Kamil, Manganaro, Lara, Pongor, Sandor, Luzzati, Roberto, Recchia, Alessandra, Mavilio, Fulvio, Giacca, Mauro, Lusic, Marina

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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements von Lattanzi, Annalisa, Meneghini, Vasco, Pavani, Giulia, Amor, Fatima, Ramadier, Sophie, Felix, Tristan, Antoniani, Chiara, Masson, Cecile, Alibeu, Olivier, Lee, Ciaran, Porteus, Matthew H., Bao, Gang, Amendola, Mario, Mavilio, Fulvio, Miccio, Annarita

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Interactions between Retroviruses and the Host Cell Genome von Poletti, Valentina, Mavilio, Fulvio

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Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication von Di Nunzio, Francesca, Fricke, Thomas, Miccio, Annarita, Valle-Casuso, Jose Carlos, Perez, Patricio, Souque, Philippe, Rizzi, Ermanno, Severgnini, Marco, Mavilio, Fulvio, Charneau, Pierre, Diaz-Griffero, Felipe

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Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts von Moiani, Arianna, Paleari, Ylenia, Sartori, Daniela, Mezzadra, Riccardo, Miccio, Annarita, Cattoglio, Claudia, Cocchiarella, Fabienne, Lidonnici, Maria Rosa, Ferrari, Giuliana, Mavilio, Fulvio

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Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair von Izmiryan, Araksya, Ganier, Clarisse, Bovolenta, Matteo, Schmitt, Alain, Mavilio, Fulvio, Hovnanian, Alain

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Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity von Eggers, Michelle, Vannoy, Charles H, Huang, Jianyong, Purushothaman, Pravinkumar, Brassard, Jacqueline, Fonck, Carlos, Meng, Hui, Prom, Mariah J, Lawlor, Michael W, Cunningham, Justine, Sadhu, Chanchal, Mavilio, Fulvio

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Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells von De Luca, Michele, Mavilio, Fulvio, Pellegrini, Graziella, Ferrari, Stefano, Di Nunzio, Francesca, Di Iorio, Enzo, Recchia, Alessandra, Maruggi, Giulietta, Ferrari, Giuliana, Provasi, Elena, Bonini, Chiara, Capurro, Sergio, Conti, Andrea, Magnoni, Cristina, Giannetti, Alberto

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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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Hot spots of retroviral integration in human CD34+ hematopoietic cells von Cattoglio, Claudia, Facchini, Giulia, Sartori, Daniela, Antonelli, Antonella, Miccio, Annarita, Cassani, Barbara, Schmidt, Manfred, von Kalle, Christof, Howe, Steve, Thrasher, Adrian J., Aiuti, Alessandro, Ferrari, Giuliana, Recchia, Alessandra, Mavilio, Fulvio

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Gene Therapy for Hemoglobinopathies von Cavazzana, Marina, Mavilio, Fulvio

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Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies von Brusson, Mégane, Chalumeau, Anne, Martinucci, Pierre, Romano, Oriana, Felix, Tristan, Poletti, Valentina, Scaramuzza, Samantha, Ramadier, Sophie, Masson, Cecile, Ferrari, Giuliana, Mavilio, Fulvio, Cavazzana, Marina, Amendola, Mario, Miccio, Annarita

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Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia von Mercado-Ayón, Elizabeth, Warren, Nathan, Halawani, Sarah, Rodden, Layne N, Ngaba, Lucie, Dong, Yi Na, Chang, Joshua C, Fonck, Carlos, Mavilio, Fulvio, Lynch, David R, Lin, Hong

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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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Transcription factor binding sites are genetic determinants of retroviral integration in the human genome von Felice, Barbara, Cattoglio, Claudia, Cittaro, Davide, Testa, Anna, Miccio, Annarita, Ferrari, Giuliana, Luzi, Lucilla, Recchia, Alessandra, Mavilio, Fulvio

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Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System von Lattanzi, Annalisa, Duguez, Stephanie, Moiani, Arianna, Izmiryan, Araksya, Barbon, Elena, Martin, Samia, Mamchaoui, Kamel, Mouly, Vincent, Bernardi, Francesco, Mavilio, Fulvio, Bovolenta, Matteo

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Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination von Coluccio, Andrea, Miselli, Francesca, Lombardo, Angelo, Marconi, Alessandra, Malagoli Tagliazucchi, Guidantonio, Gonçalves, Manuel A, Pincelli, Carlo, Maruggi, Giulietta, Del Rio, Marcela, Naldini, Luigi, Larcher, Fernando, Mavilio, Fulvio, Recchia, Alessandra

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Muscle Regeneration by Bone Marrow-Derived Myogenic Progenitors von Ferrari, Giuliana, Angelis, Gabriella Cusella-De, Coletta, Marcello, Paolucci, Egle, Stornaiuolo, Anna, Cossu, Giulio, Mavillo, Fulvio

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Muscle-Derived Hematopoietic Stem Cells are Hematopoietic in Origin von McKinney-Freeman, Shannon L., Jackson, Kathyjo A., Camargo, Fernando D., Ferrari, Giuliana, Mavilio, Fulvio, Goodell, Margaret A.

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