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	61.	Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells. [electronic resource] by Warrick, Emilie Garcia, Marta Chagnoleau, Corinne Chevallier, Odile Bergoglio, Valérie Sartori, Daniela Mavilio, Fulvio Angulo, Jaime F Avril, Marie-Françoise Sarasin, Alain Larcher, Fernando Del Rio, Marcela Bernerd, Françoise Magnaldo, Thierry Producer: 20130220 In: Molecular therapy : the journal of the American Society of Gene Therapy vol. 20 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	62.	Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements. [electronic resource] by 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 Producer: 20191211 In: Molecular therapy : the journal of the American Society of Gene Therapy vol. 27 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	63.	High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion. [electronic resource] by Cattoglio, Claudia Maruggi, Giulietta Bartholomae, Cynthia Malani, Nirav Pellin, Danilo Cocchiarella, Fabienne Magnani, Zulma Ciceri, Fabio Ambrosi, Alessandro von Kalle, Christof Bushman, Frederic D Bonini, Chiara Schmidt, Manfred Mavilio, Fulvio Recchia, Alessandra Producer: 20110705 In: PloS one vol. 5 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	64.	Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. [electronic resource] by 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 De Luca, Michele Producer: 20070222 In: Nature medicine vol. 12 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	65.	A single epidermal stem cell strategy for safe ex vivo gene therapy. [electronic resource] by Droz-Georget Lathion, Stéphanie Rochat, Ariane Knott, Graham Recchia, Alessandra Martinet, Danielle Benmohammed, Sara Grasset, Nicolas Zaffalon, Andrea Besuchet Schmutz, Nathalie Savioz-Dayer, Emmanuelle Beckmann, Jacques Samuel Rougemont, Jacques Mavilio, Fulvio Barrandon, Yann Producer: 20151221 In: EMBO molecular medicine vol. 7 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	66.	An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype. [electronic resource] by Weber, Leslie Poletti, Valentina Magrin, Elisa Antoniani, Chiara Martin, Samia Bayard, Charles Sadek, Hanem Felix, Tristan Meneghini, Vasco Antoniou, Michael N El-Nemer, Wassim Mavilio, Fulvio Cavazzana, Marina Andre-Schmutz, Isabelle Miccio, Annarita Publication details: Molecular therapy. Methods & clinical development Sep 2018 In: Molecular therapy. Methods & clinical development vol. 10 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	67.	IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors. [electronic resource] by Cieri, Nicoletta Camisa, Barbara Cocchiarella, Fabienne Forcato, Mattia Oliveira, Giacomo Provasi, Elena Bondanza, Attilio Bordignon, Claudio Peccatori, Jacopo Ciceri, Fabio Lupo-Stanghellini, Maria Teresa Mavilio, Fulvio Mondino, Anna Bicciato, Silvio Recchia, Alessandra Bonini, Chiara Producer: 20130321 In: Blood vol. 121 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	68.	Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment. [electronic resource] by Romano, Oriana Peano, Clelia Tagliazucchi, Guidantonio Malagoli Petiti, Luca Poletti, Valentina Cocchiarella, Fabienne Rizzi, Ermanno Severgnini, Marco Cavazza, Alessia Rossi, Claudia Pagliaro, Pasqualepaolo Ambrosi, Alessandro Ferrari, Giuliana Bicciato, Silvio De Bellis, Gianluca Mavilio, Fulvio Miccio, Annarita Producer: 20170308 In: Scientific reports vol. 6 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	69.	Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. [electronic resource] by Roselli, Emanuela Anna Mezzadra, Riccardo Frittoli, Marta Claudia Maruggi, Giulietta Biral, Erika Mavilio, Fulvio Mastropietro, Fabrizio Amato, Antonio Tonon, Giovanni Refaldi, Chiara Cappellini, Maria Domenica Andreani, Marco Lucarelli, Guido Roncarolo, Maria Grazia Marktel, Sarah Ferrari, Giuliana Producer: 20101110 In: EMBO molecular medicine vol. 2 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	70.	Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. [electronic resource] by Aiuti, Alessandro Cassani, Barbara Andolfi, Grazia Mirolo, Massimiliano Biasco, Luca Recchia, Alessandra Urbinati, Fabrizia Valacca, Cristina Scaramuzza, Samantha Aker, Memet Slavin, Shimon Cazzola, Matteo Sartori, Daniela Ambrosi, Alessandro Di Serio, Clelia Roncarolo, Maria Grazia Mavilio, Fulvio Bordignon, Claudio Producer: 20071004 In: The Journal of clinical investigation vol. 117 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	71.	Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia. [electronic resource] by Lidonnici, Maria Rosa Paleari, Ylenia Tiboni, Francesca Mandelli, Giacomo Rossi, Claudia Vezzoli, Michela Aprile, Annamaria Lederer, Carsten Werner Ambrosi, Alessandro Chanut, Franck Sanvito, Francesca Calabria, Andrea Poletti, Valentina Mavilio, Fulvio Montini, Eugenio Naldini, Luigi Cristofori, Patrizia Ferrari, Giuliana Publication details: Molecular therapy. Methods & clinical development Dec 2018 In: Molecular therapy. Methods & clinical development vol. 11 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	72.	Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. [electronic resource] by Antoniani, Chiara Meneghini, Vasco Lattanzi, Annalisa Felix, Tristan Romano, Oriana Magrin, Elisa Weber, Leslie Pavani, Giulia El Hoss, Sara Kurita, Ryo Nakamura, Yukio Cradick, Thomas J Lundberg, Ante S Porteus, Matthew Amendola, Mario El Nemer, Wassim Cavazzana, Marina Mavilio, Fulvio Miccio, Annarita Producer: 20190514 In: Blood vol. 131 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	73.	Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning. [electronic resource] by Huston, Marshall W van Til, Niek P Visser, Trudi P Arshad, Shazia Brugman, Martijn H Cattoglio, Claudia Nowrouzi, Ali Li, Yuedan Schambach, Axel Schmidt, Manfred Baum, Christopher von Kalle, Christof Mavilio, Fulvio Zhang, Fang Blundell, Mike P Thrasher, Adrian J Verstegen, Monique M A Wagemaker, Gerard Producer: 20120203 In: Molecular therapy : the journal of the American Society of Gene Therapy vol. 19 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	74.	Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors. [electronic resource] by Holstein, Marta Mesa-Nuñez, Cristina Miskey, Csaba Almarza, Elena Poletti, Valentina Schmeer, Marco Grueso, Esther Ordóñez Flores, Juan Carlos Kobelt, Dennis Walther, Wolfgang Aneja, Manish K Geiger, Johannes Bonig, Halvard B Izsvák, Zsuzsanna Schleef, Martin Rudolph, Carsten Mavilio, Fulvio Bueren, Juan A Guenechea, Guillermo Ivics, Zoltán Producer: 20190423 In: Molecular therapy : the journal of the American Society of Gene Therapy vol. 26 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	75.	Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination. [electronic resource] by André-Schmutz, Isabelle Dal-Cortivo, Liliane Six, Emmanuelle Kaltenbach, Sophie Cocchiarella, Fabienne Le Chenadec, Jerome Cagnard, Nicolas Cordier, Anne-Gael Benachi, Alexandra Mandelbrot, Laurent Azria, Elie Bouallag, Naima Luce, Sonia Ternaux, Brigitte Reimann, Christian Revy, Patrick Radford-Weiss, Isabelle Leschi, Cristina Recchia, Alessandra Mavilio, Fulvio Cavazzana, Marina Blanche, Stéphane Producer: 20131113 In: The Journal of infectious diseases vol. 208 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	76.	Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. [electronic resource] by Morris, Emma C Fox, Thomas Chakraverty, Ronjon Tendeiro, Rita Snell, Katie Rivat, Christine Grace, Sarah Gilmour, Kimberly Workman, Sarita Buckland, Karen Butler, Katie Chee, Ronnie Salama, Alan D Ibrahim, Hazem Hara, Havinder Duret, Cecile Mavilio, Fulvio Male, Frances Bushman, Frederick D Galy, Anne Burns, Siobhan O Gaspar, H Bobby Thrasher, Adrian J Producer: 20171002 In: Blood vol. 130 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	77.	Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. [electronic resource] by Deichmann, Annette Brugman, Martijn H Bartholomae, Cynthia C Schwarzwaelder, Kerstin Verstegen, Monique M A Howe, Steven J Arens, Anne Ott, Marion G Hoelzer, Dieter Seger, Reinhard Grez, Manuel Hacein-Bey-Abina, Salima Cavazzana-Calvo, Marina Fischer, Alain Paruzynski, Anna Gabriel, Richard Glimm, Hanno Abel, Ulrich Cattoglio, Claudia Mavilio, Fulvio Cassani, Barbara Aiuti, Alessandro Dunbar, Cynthia E Baum, Christopher Gaspar, H Bobby Thrasher, Adrian J von Kalle, Christof Schmidt, Manfred Wagemaker, Gerard Producer: 20120305 In: Molecular therapy : the journal of the American Society of Gene Therapy vol. 19 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	78.	Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. [electronic resource] by Childers, Martin K Joubert, Romain Poulard, Karine Moal, Christelle Grange, Robert W Doering, Jonathan A Lawlor, Michael W Rider, Branden E Jamet, Thibaud Danièle, Nathalie Martin, Samia Rivière, Christel Soker, Thomas Hammer, Caroline Van Wittenberghe, Laetitia Lockard, Mandy Guan, Xuan Goddard, Melissa Mitchell, Erin Barber, Jane Williams, J Koudy Mack, David L Furth, Mark E Vignaud, Alban Masurier, Carole Mavilio, Fulvio Moullier, Philippe Beggs, Alan H Buj-Bello, Anna Producer: 20140910 In: Science translational medicine vol. 6 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	79.	Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. [electronic resource] by Mack, David L Poulard, Karine Goddard, Melissa A Latournerie, Virginie Snyder, Jessica M Grange, Robert W Elverman, Matthew R Denard, Jérôme Veron, Philippe Buscara, Laurine Le Bec, Christine Hogrel, Jean-Yves Brezovec, Annie G Meng, Hui Yang, Lin Liu, Fujun O'Callaghan, Michael Gopal, Nikhil Kelly, Valerie E Smith, Barbara K Strande, Jennifer L Mavilio, Fulvio Beggs, Alan H Mingozzi, Federico Lawlor, Michael W Buj-Bello, Ana Childers, Martin K Producer: 20170606 In: Molecular therapy : the journal of the American Society of Gene Therapy vol. 25 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	80.	Comprehensive genomic access to vector integration in clinical gene therapy. [electronic resource] by Gabriel, Richard Eckenberg, Ralph Paruzynski, Anna Bartholomae, Cynthia C Nowrouzi, Ali Arens, Anne Howe, Steven J Recchia, Alessandra Cattoglio, Claudia Wang, Wei Faber, Katrin Schwarzwaelder, Kerstin Kirsten, Romy Deichmann, Annette Ball, Claudia R Balaggan, Kamaljit S Yáñez-Muñoz, Rafael J Ali, Robin R Gaspar, H Bobby Biasco, Luca Aiuti, Alessandro Cesana, Daniela Montini, Eugenio Naldini, Luigi Cohen-Haguenauer, Odile Mavilio, Fulvio Thrasher, Adrian J Glimm, Hanno von Kalle, Christof Saurin, William Schmidt, Manfred Producer: 20091215 In: Nature medicine vol. 15 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)

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