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22 results on '"Plati, Tiziana"'

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

Author

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, and Cantore, Alessio

Published
2022
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2. P1405: IN-DEPTH GENOME INTEGRITY EVALUATION AND GMP-COMPLIANT MANUFACTURING OF HDR GENE EDITED CD4+ T CELLS FOR THE TREATMENT OF HYPER IGM 1

Author

Canarutto, Daniele, primary, Asperti, Claudia, additional, Vavassori, Valentina, additional, Porcellini, Simona, additional, Rovelli, Elisabetta, additional, Paulis, Marianna, additional, Ferrari, Samuele, additional, Plati, Tiziana, additional, Sergi Sergi, Lucia, additional, Villa, Anna, additional, Radrizzani, Marina, additional, and Naldini, Luigi, additional

Published
2023
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4. MicroRNA-223 dose levels fine tune proliferation and differentiation in human cord blood progenitors and acute myeloid leukemia

Author

Gentner, Bernhard, Pochert, Nicole, Rouhi, Arefeh, Boccalatte, Francesco, Plati, Tiziana, Berg, Tobias, Sun, Su Ming, Mah, Sarah M., Mirkovic-Hösle, Milijana, Ruschmann, Jens, Muranyi, Andrew, Leierseder, Simon, Argiropoulos, Bob, Starczynowski, Daniel T., Karsan, Aly, Heuser, Michael, Hogge, Donna, Camargo, Fernando D., Engelhardt, Stefan, Döhner, Hartmut, Buske, Christian, Jongen-Lavrencic, Mojca, Naldini, Luigi, Humphries, R. Keith, and Kuchenbauer, Florian

Published
2015
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5. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Author

Schiroli, Giulia, Ferrari, Samuele, Conway, Anthony, Jacob, Aurelien, Capo, Valentina, Albano, Luisa, Plati, Tiziana, Castiello, Maria C., Sanvito, Francesca, Gennery, Andrew R., Bovolenta, Chiara, Palchaudhuri, Rahul, Scadden, David T., Holmes, Michael C., Villa, Anna, Sitia, Giovanni, Lombardo, Angelo, Genovese, Pietro, and Naldini, Luigi

Published
2017
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6. Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Author

Biffi, Alessandra, Montini, Eugenio, Lorioli, Laura, Cesani, Martina, Fumagalli, Francesca, Plati, Tiziana, Baldoli, Cristina, Martino, Sabata, Calabria, Andrea, Canale, Sabrina, Benedicenti, Fabrizio, Vallanti, Giuliana, Biasco, Luca, Leo, Simone, Kabbara, Nabil, Zanetti, Gianluigi, Rizzo, William B., Mehta, Nalini A. L., Cicalese, Maria Pia, Casiraghi, Miriam, Boelens, Jaap J., Del Carro, Ubaldo, Dow, David J., Schmidt, Manfred, Assanelli, Andrea, Neduva, Victor, Di Serio, Clelia, Stupka, Elia, Gardner, Jason, von Kalle, Christof, Bordignon, Claudio, Ciceri, Fabio, Rovelli, Attilio, Roncarolo, Maria Grazia, Aiuti, Alessandro, Sessa, Maria, and Naldini, Luigi

Published
2013

7. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

Author

Biffi, Alessandra, Bartolomae, Cynthia C., Cesana, Daniela, Cartier, Natalie, Aubourg, Patrik, Ranzani, Marco, Cesani, Martina, Benedicenti, Fabrizio, Plati, Tiziana, Rubagotti, Enrico, Merella, Stefania, Capotondo, Alessia, Sgualdino, Jacopo, Zanetti, Gianluigi, von Kalle, Christof, Schmidt, Manfred, Naldini, Luigi, and Montini, Eugenio

Published
2011
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8. Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

Author

Milani, Michela, primary, Canepari, Cesare, additional, Liu, Tongyao, additional, Biffi, Mauro, additional, Russo, Fabio, additional, Plati, Tiziana, additional, Curto, Rosalia, additional, Patarroyo-White, Susannah, additional, Drager, Douglas, additional, Visigalli, Ilaria, additional, Brombin, Chiara, additional, Albertini, Paola, additional, Ayuso, Eduard, additional, Mueller, Christian, additional, Annoni, Andrea, additional, Naldini, Luigi, additional, and Cantore, Alessio, additional

Published
2021
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13. Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

Author

Escobar, Giulia, primary, Barbarossa, Luigi, additional, Barbiera, Giulia, additional, Norelli, Margherita, additional, Genua, Marco, additional, Ranghetti, Anna, additional, Plati, Tiziana, additional, Camisa, Barbara, additional, Brombin, Chiara, additional, Cittaro, Davide, additional, Annoni, Andrea, additional, Bondanza, Attilio, additional, Ostuni, Renato, additional, Gentner, Bernhard, additional, and Naldini, Luigi, additional

Published
2018
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14. 27. Aberrant Expression of the Stem Cell microRNA-126 Induces B Cell Malignancy

Author

Nucera, Silvia, primary, Boccalatte, Francesco, additional, Calabria, Andrea, additional, Plati, Tiziana, additional, Fanciullo, Cristiana, additional, Manteiga, Jose, additional, Benedicenti, Fabrizio, additional, Ciceri, Fabio, additional, Ponzoni, Maurilio, additional, Montini, Eugenio, additional, Naldini, Luigi, additional, and Gentner, Bernhard, additional

Published
2015
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17. A Mechanistic Role For Mir-126, a Hematopoietic Stem Cell Microrna, In Acute Leukemias

Author

Giustacchini, Alice, primary, Nucera, Silvia, additional, Lechman, Eric R., additional, Fanciullo, Cristiana, additional, Boccalatte, Francesco E, additional, Plati, Tiziana, additional, Ranghetti, Anna, additional, Vago, Luca, additional, Bernardi, Massimo, additional, Ponzoni, Maurilio, additional, Dick, John E., additional, Naldini, Luigi, additional, and Gentner, Bernhard, additional

Published
2013
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18. Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

Author

Capotondo, Alessia, primary, Milazzo, Rita, additional, Politi, Letterio Salvatore, additional, Quattrini, Angelo, additional, Palini, Alessio, additional, Plati, Tiziana, additional, Merella, Stefania, additional, Nonis, Alessandro, additional, di Serio, Clelia, additional, Montini, Eugenio, additional, Naldini, Luigi, additional, and Biffi, Alessandra, additional

Published
2012
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20. 9. Hematopoietic stem cell based gene therapy for the treatment of metachromatic leukodystrophy: Towards clinical testing

Author

Biffi, Alessandra, primary, Capotondo, Alessia, additional, Cesani, Martina, additional, Montini, Eugenio, additional, Plati, Tiziana, additional, Benedicenti, Fabrizio, additional, Tononi, Laura, additional, Marchesini, Sergio, additional, Sessa, Maria, additional, Bordignon, Claudio, additional, Roncarolo, Maria Grazia, additional, and Naldini, Luigi, additional

Published
2008
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21. miRNA-126 Orchestrates an Oncogenic Program in B Cell Precursor Acute Lymphoblastic Leukemia

Author

Francesco Boccalatte, Anna Ranghetti, Fabio Ciceri, Eric R. Lechman, Jose Manuel Garcia-Manteiga, Bernhard Gentner, Luigi Naldini, Silvia Nucera, Maurilio Ponzoni, Tiziana Plati, Fabrizio Benedicenti, Eugenio Montini, Andrea Calabria, Alice Giustacchini, Davide Cittaro, Cristiana Fanciullo, John E. Dick, Nucera, Silvia, Giustacchini, Alice, Boccalatte, Francesco, Calabria, Andrea, Fanciullo, Cristiana, Plati, Tiziana, Ranghetti, Anna, Garcia Manteiga, Jose, Cittaro, Davide, Benedicenti, Fabrizio, Lechman, Eric R., Dick, John E., Ponzoni, Maurilio, Ciceri, Fabio, Montini, Eugenio, Gentner, Bernhard, and Naldini, Luigi

Subjects
0301 basic medicine, Cancer Research, Cellular differentiation, Apoptosis, Biology, Mice, 03 medical and health sciences, Downregulation and upregulation, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, microRNA, medicine, Animals, Humans, Progenitor cell, B cell, Regulation of gene expression, Cell Cycle, Hematopoietic Stem Cell Transplantation, Cell Differentiation, Neoplasms, Experimental, Cell Biology, Cell cycle, Hematopoietic Stem Cells, medicine.disease, Up-Regulation, Gene Expression Regulation, Neoplastic, MicroRNAs, 030104 developmental biology, medicine.anatomical_structure, Oncology, B-cell leukemia, Cancer research, Tumor Suppressor Protein p53, Signal Transduction
Abstract

MicroRNA (miRNA)-126 is a known regulator of hematopoietic stem cell quiescence. We engineered murine hematopoiesis to express miRNA-126 across all differentiation stages. Thirty percent of mice developed monoclonal B cell leukemia, which was prevented or regressed when a tetracycline-repressible miRNA-126 cassette was switched off. Regression was accompanied by upregulation of cell-cycle regulators and B cell differentiation genes, and downregulation of oncogenic signaling pathways. Expression of dominant-negative p53 delayed blast clearance upon miRNA-126 switch-off, highlighting the relevance of p53 inhibition in miRNA-126 addiction. Forced miRNA-126 expression in mouse and human progenitors reduced p53 transcriptional activity through regulation of multiple p53-related targets. miRNA-126 is highly expressed in a subset of human B-ALL, and antagonizing miRNA-126 in ALL xenograft models triggered apoptosis and reduced disease burden.

Published
2016
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22. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Author

Giulia Schiroli, Anthony Conway, Samuele Ferrari, Michael C. Holmes, Luigi Naldini, Maria Carmina Castiello, Rahul Palchaudhuri, Anna Villa, Aurelien Jacob, Francesca Sanvito, Angelo Lombardo, Valentina Capo, Tiziana Plati, David T. Scadden, Chiara Bovolenta, Luisa Albano, Pietro Genovese, Giovanni Sitia, Andrew R. Gennery, Schiroli, Giulia, Ferrari, Samuele, Conway, Anthony, Jacob, Aurelien, Capo, Valentina, Albano, Luisa, Plati, Tiziana, Castiello, Maria C., Sanvito, Francesca, Gennery, Andrew R., Bovolenta, Chiara, Palchaudhuri, Rahul, Scadden, David T., Holmes, Michael C., Villa, Anna, Sitia, Giovanni, Lombardo, Angelo, Genovese, Pietro, and Naldini, Luigi

Subjects
0301 basic medicine, Mice, SCID, Computational biology, Biology, Bioinformatics, Mice, 03 medical and health sciences, Genome editing, medicine, Animals, CRISPR, Clustered Regularly Interspaced Short Palindromic Repeats, Progenitor cell, Gene Editing, Severe combined immunodeficiency, Cas9, Medicine (all), Hematopoietic stem cell, General Medicine, Hematopoietic Stem Cells, medicine.disease, Zinc finger nuclease, Haematopoiesis, 030104 developmental biology, medicine.anatomical_structure, Gene Targeting, Interleukin Receptor Common gamma Subunit
Abstract

Targeted genome editing in hematopoietic stem/progenitor cells (HSPCs) is an attractive strategy for treating immunohematological diseases. However, the limited efficiency of homology-directed editing in primitive HSPCs constrains the yield of corrected cells and might affect the feasibility and safety of clinical translation. These concerns need to be addressed in stringent preclinical models and overcome by developing more efficient editing methods. We generated a humanized X-linked severe combined immunodeficiency (SCID-X1) mouse model and evaluated the efficacy and safety of hematopoietic reconstitution from limited input of functional HSPCs, establishing thresholds for full correction upon different types of conditioning. Unexpectedly, conditioning before HSPC infusion was required to protect the mice from lymphoma developing when transplanting small numbers of progenitors. We then designed a one-size-fits-all IL2RG (interleukin-2 receptor common γ-chain) gene correction strategy and, using the same reagents suitable for correction of human HSPC, validated the edited human gene in the disease model in vivo, providing evidence of targeted gene editing in mouse HSPCs and demonstrating the functionality of the IL2RG-edited lymphoid progeny. Finally, we optimized editing reagents and protocol for human HSPCs and attained the threshold of IL2RG editing in long-term repopulating cells predicted to safely rescue the disease, using clinically relevant HSPC sources and highly specific zinc finger nucleases or CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 (CRISPR-associated protein 9). Overall, our work establishes the rationale and guiding principles for clinical translation of SCID-X1 gene editing and provides a framework for developing gene correction for other diseases.

Published
2017

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