Pesquisa | Biblioteca Virtual em Saúde

1.

Nuclear architecture dictates HIV-1 integration site selection.

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.

Nature; 521(7551): 227-31, 2015 May 14.

Artigo em Inglês | MEDLINE | ID: mdl-25731161

2.

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human ß-globin locus.

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.

Blood; 131(17): 1960-1973, 2018 Apr 26.

Artigo em Inglês | MEDLINE | ID: mdl-29519807

3.

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.

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.

Blood; 130(11): 1327-1335, 2017 09 14.

Artigo em Inglês | MEDLINE | ID: mdl-28716862

4.

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.

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.

Mol Ther; 2018 Oct 17.

Artigo em Inglês | MEDLINE | ID: mdl-30424953

5.

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.

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.

Mol Ther; 26(4): 1137-1153, 2018 Apr 04.

Artigo em Inglês | MEDLINE | ID: mdl-29503198

6.

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.

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.

Mol Ther; 25(4): 839-854, 2017 04 05.

Artigo em Inglês | MEDLINE | ID: mdl-28237839

7.

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.

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.

Blood; 121(4): 573-84, 2013 Jan 24.

Artigo em Inglês | MEDLINE | ID: mdl-23160470

8.

The future of gene therapy.

Cavazzana-Calvo, Marina; Thrasher, Adrian; Mavilio, Fulvio.

Nature; 427(6977): 779-81, 2004 Feb 26.

Artigo em Inglês | MEDLINE | ID: mdl-14985734

9.

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Hacein-Bey Abina, Salima; Gaspar, H Bobby; Blondeau, Johanna; Caccavelli, Laure; Charrier, Sabine; Buckland, Karen; Picard, Capucine; Six, Emmanuelle; Himoudi, Nourredine; Gilmour, Kimberly; McNicol, Anne-Marie; Hara, Havinder; Xu-Bayford, Jinhua; Rivat, Christine; Touzot, Fabien; Mavilio, Fulvio; Lim, Annick; Treluyer, Jean-Marc; Héritier, Sébastien; Lefrère, Francois; Magalon, Jeremy; Pengue-Koyi, Isabelle; Honnet, Géraldine; Blanche, Stéphane; Sherman, Eric A; Male, Frances; Berry, Charles; Malani, Nirav; Bushman, Frederic D; Fischer, Alain; Thrasher, Adrian J; Galy, Anne; Cavazzana, Marina.

JAMA; 313(15): 1550-63, 2015 Apr 21.

Artigo em Inglês | MEDLINE | ID: mdl-25898053

10.

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.

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.

Mol Ther; 21(9): 1695-704, 2013 Sep.

Artigo em Inglês | MEDLINE | ID: mdl-23760447

11.

Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination.

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.

J Infect Dis; 208(2): 235-43, 2013 Jul 15.

Artigo em Inglês | MEDLINE | ID: mdl-23559464

12.

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.

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.

Nat Med; 12(12): 1397-402, 2006 Dec.

Artigo em Inglês | MEDLINE | ID: mdl-17115047

13.

Gene therapies need new development models.

Mavilio, Fulvio.

Nature; 490(7418): 7, 2012 Oct 04.

Artigo em Inglês | MEDLINE | ID: mdl-23038429

14.

Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.

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.

Mol Ther; 20(4): 798-807, 2012 Apr.

Artigo em Inglês | MEDLINE | ID: mdl-22068429

15.

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.

Cattoglio, Claudia; Pellin, Danilo; Rizzi, Ermanno; Maruggi, Giulietta; Corti, Giorgio; Miselli, Francesca; Sartori, Daniela; Guffanti, Alessandro; Di Serio, Clelia; Ambrosi, Alessandro; De Bellis, Gianluca; Mavilio, Fulvio.

Blood; 116(25): 5507-17, 2010 Dec 16.

Artigo em Inglês | MEDLINE | ID: mdl-20864581

16.

C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cells.

Barbaro, Vanessa; Testa, Anna; Di Iorio, Enzo; Mavilio, Fulvio; Pellegrini, Graziella; De Luca, Michele.

J Cell Biol; 177(6): 1037-49, 2007 Jun 18.

Artigo em Inglês | MEDLINE | ID: mdl-17562792

17.

Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors.

Ambrosi, Alessandro; Glad, Ingrid K; Pellin, Danilo; Cattoglio, Claudia; Mavilio, Fulvio; Di Serio, Clelia; Frigessi, Arnoldo.

PLoS Comput Biol; 7(12): e1002292, 2011 Dec.

Artigo em Inglês | MEDLINE | ID: mdl-22144885

18.

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.

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.

Mol Ther; 19(10): 1867-77, 2011 Oct.

Artigo em Inglês | MEDLINE | ID: mdl-21750532

19.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.

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.

Mol Ther; 19(11): 2031-9, 2011 Nov.

Artigo em Inglês | MEDLINE | ID: mdl-21862999

20.

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Cassani, Barbara; Montini, Eugenio; Maruggi, Giulietta; Ambrosi, Alessandro; Mirolo, Massimiliano; Selleri, Silvia; Biral, Erika; Frugnoli, Ilaria; Hernandez-Trujillo, Vivian; Di Serio, Clelia; Roncarolo, Maria Grazia; Naldini, Luigi; Mavilio, Fulvio; Aiuti, Alessandro.

Blood; 114(17): 3546-56, 2009 Oct 22.

Artigo em Inglês | MEDLINE | ID: mdl-19652199

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