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ArticlesPeer ReviewedFull Text
Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives
Author / Creator:Naldini, Luigi
Publisher:London: Nature Publishing Group UK
Journal title:EMBO molecular medicine, 2019-03, Vol.11 (3), p.1-n/a
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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
Author / Creator:Tucci, Francesca ; Galimberti, Stefania ; Naldini, Luigi ; Valsecchi, Maria Grazia ; Aiuti, Alessandro
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2022-03, Vol.13 (1), p.1315-13, Article 1315
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Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products
Author / Creator:Aiuti, Alessandro ; Roncarolo, Maria Grazia ; Naldini, Luigi
Publisher:London: Nature Publishing Group UK
Journal title:EMBO molecular medicine, 2017-06, Vol.9 (6), p.737-740
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Ensuring a future for gene therapy for rare diseases
Author / Creator:Aiuti, Alessandro ; Pasinelli, Francesca ; Naldini, Luigi
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2022-10, Vol.28 (10), p.1985-1988
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Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking
Author / Creator:Ferrari, Samuele ; Jacob, Aurelien ; Beretta, Stefano ; Unali, Giulia ; Albano, Luisa ; Vavassori, Valentina ; Cittaro, Davide ; Lazarevic, Dejan ; Brombin, Chiara ; Cugnata, Federica ; Kajaste-Rudnitski, Anna ; Merelli, Ivan ; Genovese, Pietro ; Naldini, Luigi
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2020-11, Vol.38 (11), p.1298-1308
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ArticlesPeer ReviewedFull Text
A Comeback for Gene Therapy
Author / Creator:Naldini, Luigi
Publisher:Washington: American Association for the Advancement of Science
Journal title:Science (American Association for the Advancement of Science), 2009-11, Vol.326 (5954), p.805-806
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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications
Author / Creator:Brown, Brian D. ; Naldini, Luigi
Publisher:London: Nature Publishing Group UK
Journal title:Nature reviews. Genetics, 2009-08, Vol.10 (8), p.578-585
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Genotoxic effects of base and prime editing in human hematopoietic stem cells
Author / Creator:Fiumara, Martina ; Ferrari, Samuele ; Omer-Javed, Attya ; Beretta, Stefano ; Albano, Luisa ; Canarutto, Daniele ; Varesi, Angelica ; Gaddoni, Chiara ; Brombin, Chiara ; Cugnata, Federica ; Zonari, Erika ; Naldini, Matteo Maria ; Barcella, Matteo ; Gentner, Bernhard ; Merelli, Ivan ; Naldini, Luigi
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2024-06, Vol.42 (6), p.877-891
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ArticlesPeer ReviewedFull Text
Adopt a moratorium on heritable genome editing
Author / Creator:Lander, Eric S. ; Baylis, Françoise ; Zhang, Feng ; Charpentier, Emmanuelle ; Berg, Paul ; Bourgain, Catherine ; Friedrich, Bärbel ; Joung, J. Keith ; Li, Jinsong ; Liu, David ; Naldini, Luigi ; Nie, Jing-Bao ; Qiu, Renzong ; Schoene-Seifert, Bettina ; Shao, Feng ; Terry, Sharon ; Wei, Wensheng ; Winnacker, Ernst-Ludwig
Publisher:London: Nature Publishing Group UK
Journal title:Nature (London), 2019-03, Vol.567 (7747), p.165-168
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Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
Author / Creator:Milani, Michela ; Canepari, Cesare ; Liu, Tongyao ; Biffi, Mauro ; Russo, Fabio ; Plati, Tiziana ; Curto, Rosalia ; Patarroyo-White, Susannah ; Drager, Douglas ; Visigalli, Ilaria ; Brombin, Chiara ; Albertini, Paola ; Follenzi, Antonia ; Ayuso, Eduard ; Mueller, Christian ; Annoni, Andrea ; Naldini, Luigi ; Cantore, Alessio
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2022-05, Vol.13 (1), p.2454-2454, Article 2454
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ArticlesPeer ReviewedFull Text
BAR-Seq clonal tracking of gene-edited cells
Author / Creator:Ferrari, Samuele ; Beretta, Stefano ; Jacob, Aurelien ; Cittaro, Davide ; Albano, Luisa ; Merelli, Ivan ; Naldini, Luigi ; Genovese, Pietro
Publisher:London: Nature Publishing Group UK
Journal title:Nature protocols, 2021-06, Vol.16 (6), p.2991-3025
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ArticlesPeer ReviewedFull Text
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations
Author / Creator:Cesana, Daniela ; Sgualdino, Jacopo ; Rudilosso, Laura ; Merella, Stefania ; Naldini, Luigi ; Montini, Eugenio
Publisher:United States: American Society for Clinical Investigation
Journal title:The Journal of clinical investigation, 2012-05, Vol.122 (5), p.1667-1676
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ArticlesPeer ReviewedFull Text
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
Author / Creator:Montini, Eugenio ; Cesana, Daniela ; Schmidt, Manfred ; Sanvito, Francesca ; Bartholomae, Cynthia C. ; Ranzani, Marco ; Benedicenti, Fabrizio ; Sergi, Lucia Sergi ; Ambrosi, Alessandro ; Ponzoni, Maurilio ; Doglioni, Claudio ; Di Serio, Clelia ; von Kalle, Christof ; Naldini, Luigi
Publisher:United States: American Society for Clinical Investigation
Journal title:The Journal of clinical investigation, 2009-04, Vol.119 (4), p.964-975
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ArticlesPeer ReviewedFull Text
Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation
Author / Creator:Capotondo, Alessia ; Milazzo, Rita ; Politi, Letterio Salvatore ; Quattrini, Angelo ; Palini, Alessio ; Plati, Tiziana ; Merella, Stefania ; Nonis, Alessandro ; di Serio, Clelia ; Montini, Eugenio ; Naldini, Luigi ; Biffi, Alessandra
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2012-09, Vol.109 (37), p.15018-15023
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ArticlesPeer ReviewedFull Text
Retrieval of vector integration sites from cell-free DNA
Author / Creator:Cesana, Daniela ; Calabria, Andrea ; Rudilosso, Laura ; Gallina, Pierangela ; Benedicenti, Fabrizio ; Spinozzi, Giulio ; Schiroli, Giulia ; Magnani, Alessandra ; Acquati, Serena ; Fumagalli, Francesca ; Calbi, Valeria ; Witzel, Maximilian ; Bushman, Frederic D. ; Cantore, Alessio ; Genovese, Pietro ; Klein, Christoph ; Fischer, Alain ; Cavazzana, Marina ; Six, Emmanuelle ; Aiuti, Alessandro ; Naldini, Luigi ; Montini, Eugenio
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2021-08, Vol.27 (8), p.1458-1470
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ArticlesPeer ReviewedFull Text
Site-specific integration and tailoring of cassette design for sustainable gene transfer
Author / Creator:Lombardo, Angelo ; Cesana, Daniela ; Genovese, Pietro ; Di Stefano, Bruno ; Provasi, Elena ; Colombo, Daniele F ; Neri, Margherita ; Magnani, Zulma ; Cantore, Alessio ; Lo Riso, Pietro ; Damo, Martina ; Pello, Oscar M ; Holmes, Michael C ; Gregory, Philip D ; Gritti, Angela ; Broccoli, Vania ; Bonini, Chiara ; Naldini, Luigi
Publisher:New York: Nature Publishing Group US
Journal title:Nature methods, 2011-10, Vol.8 (10), p.861-869
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ArticlesPeer ReviewedFull Text
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
Author / Creator:Lombardo, Angelo ; Genovese, Pietro ; Beausejour, Christian M ; Colleoni, Silvia ; Lee, Ya-Li ; Kim, Kenneth A ; Ando, Dale ; Urnov, Fyodor D ; Galli, Cesare ; Gregory, Philip D ; Holmes, Michael C ; Naldini, Luigi
Publisher:New York: Nature Publishing Group US
Journal title:Nature biotechnology, 2007-11, Vol.25 (11), p.1298-1306
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ArticlesPeer ReviewedFull Text
Gene therapy returns to centre stage
Author / Creator:Naldini, Luigi
Publisher:London: Nature Publishing Group UK
Journal title:Nature (London), 2015-10, Vol.526 (7573), p.351-360
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Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens
Author / Creator:Escobar, Giulia ; Barbarossa, Luigi ; Barbiera, Giulia ; Norelli, Margherita ; Genua, Marco ; Ranghetti, Anna ; Plati, Tiziana ; Camisa, Barbara ; Brombin, Chiara ; Cittaro, Davide ; Annoni, Andrea ; Bondanza, Attilio ; Ostuni, Renato ; Gentner, Bernhard ; Naldini, Luigi
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2018-07, Vol.9 (1), p.2896-16, Article 2896
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TIE2-expressing macrophages limit the therapeutic efficacy of the vascular-disrupting agent combretastatin A4 phosphate in mice
Author / Creator:Welford, Abigail F. ; Biziato, Daniela ; Coffelt, Seth B. ; Nucera, Silvia ; Fisher, Matthew ; Pucci, Ferdinando ; Di Serio, Clelia ; Naldini, Luigi ; De Palma, Michele ; Tozer, Gillian M. ; Lewis, Claire E.
Publisher:United States: American Society for Clinical Investigation
Journal title:The Journal of clinical investigation, 2011-05, Vol.121 (5), p.1969-1973
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Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives

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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

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Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

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Ensuring a future for gene therapy for rare diseases

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Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

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A Comeback for Gene Therapy

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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

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Genotoxic effects of base and prime editing in human hematopoietic stem cells

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Adopt a moratorium on heritable genome editing

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Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

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BAR-Seq clonal tracking of gene-edited cells

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Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations

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The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

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Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

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Retrieval of vector integration sites from cell-free DNA

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Site-specific integration and tailoring of cassette design for sustainable gene transfer

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

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Gene therapy returns to centre stage

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Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

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TIE2-expressing macrophages limit the therapeutic efficacy of the vascular-disrupting agent combretastatin A4 phosphate in mice

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