Sfoglia per Autore
Perspective on innovative therapies for globoid cell leukodystrophy
2016-01-01 Ricca, A.; Gritti, A.
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy
2016-01-01 Meneghini, Vasco; Lattanzi, Annalisa; Tiradani, Luigi; Bravo, Gabriele; Morena, Francesco; Sanvito, Francesca; Calabria, Andrea; Bringas, John; Fisher-Perkins, Jeanne M; Dufour, Jason P; Baker, Kate C; Doglioni, Claudio; Montini, Eugenio; Bunnell, Bruce A; Bankiewicz, Krystof; Martino, Sabata; Naldini, Luigi; Gritti, Angela
Early sulfatide storage and progressive neuronal and glial cell dysfunction revealed by human iPSC-based models of metachromatic leukodystrophy are rescued by lentiviral-mediated restoration of ARSA activity
2017-01-01 Frati, ; G, ; Luciani, ; M, ; Meneghini, ; V, ; De, Cicco; S, ; Stahlman, ; M, ; Grossi, ; S, ; Morena, ; F, ; Blomqvist, ; M, ; Filocamo, ; M, ; Martino, ; S, ; Gritti, A
Genome-Wide Definition of Regulatory Regions and Transcripts During the Transition from Pluripotent to Neural-Restricted Stem Cells
2017-01-01 Meneghini, ; V, ; Luciani, ; M, ; Petiti, ; L, ; Cifola, ; I, ; Peano, ; C, ; Gritti, A; Miccio, A
Neuronal and glial cells derived from a murine model of GM2-gangliosidosis display typical hallmarks of pathology and represent a valuable model to test gene therapy strategies
2017-01-01 Ornaghi, ; F, ; Tedeschi, ; F, ; Sala, ; D, ; Ricca, ; A, ; Di, Tomaso; T, ; Morena, ; F, ; Martino, ; S, ; Gritti, A
Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy
2017-01-01 Meneghini, Vasco; Frati, Giacomo; Sala, Davide; De Cicco, Silvia; Luciani, Marco; Cavazzin, Chiara; Paulis, Marianna; Mentzen, Wieslawa; Morena, Francesco; Giannelli, Serena; Sanvito, Francesca; Villa, Anna; Bulfone, Alessandro; Broccoli, Vania; Martino, Sabata; Gritti, Angela
Two factor-based reprogramming of rodent and human fibroblasts into Schwann cells
2017-01-01 Mazzara, P. G.; Massimino, L; Pellegatta, M; Ronchi, G; Ricca, A; Iannielli, A; Giannelli, S. G.; Cursi, M; Cancellieri, C; Sessa, A; Del Carro, U; Quattrini, A; Geuna, S; Gritti, A; Taveggia, C; Broccoli, V.
Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy
2018-01-01 Frati, Giacomo; Luciani, Marco; Meneghini, Vasco; De Cicco, Silvia; Ståhlman, Marcus; Blomqvist, Maria; Grossi, Serena; Filocamo, Mirella; Morena, Francesco; Menegon, Andrea; Martino, Sabata; Gritti, Angela
Optimized bicistronic lentiviral vectors to correct b-hexosaminidase deficiency in neural and hematopoietic stem/progenitor cells and progeny
2019-01-01 Ornaghi, F; Sala, D; Tedeschi, F; Bazzucchi, M; Morena, F; Martino, S; Gritti, A
Integrated computational analysis highlights unique miRNA signatures in the subventricular zone and striatum of GM2 gangliosidosis animal models
2019-01-01 Morena, F; Oikonomou, V; Argentati, C; Bazzucchi, M; Emiliani, C; Gritti, A; Martino, S.
Genome-wide analyses of regulatory regions and transcripts in hiPSC-derived neural stem/progenitor cells to define their safety and efficacy in cell therapy approaches for neurological disorders
2019-01-01 Meneghini, V; Luciani, M; Petiti, L; Cifola, I; Peano, C; Miccio, A; Gritti, A
Exploiting targeted epigenetic editing to increase the yield, homogeneity and purity of human iPSCderived oligodendrocyte cell populations
2019-01-01 Luciani, M; Ciccarelli, E; Migliara, A; Lombardo, A; Gritti, A
In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion
2020-01-01 Ricca, A; Cascino, F; Morena, F; Martino, S; Gritti, A.
Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis
2020-01-01 Ornaghi, F; Sala, D; Tedeschi, F; Maffia, M. C.; Bazzucchi, M; Morena, F; Valsecchi, M; Aureli, M; Martino, S; Gritti, A.
Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases
2020-01-01 Luciani, Marco; Gritti, Angela; Meneghini, Vasco
Chimeric Lysosomal Enzymes with Improved Bioavailability to Advance Gene Therapy Strategies for Globoid Cell Leukodystrophy
2021-01-01 Cascino, F; Ricca, A; Morena, F; Martino, S; Gritti, A
A Human IPSC-Based Model of Globoid Cell Leukodystrophy Uncovers Early Neurodevelopmental Defects
2021-01-01 Mangiameli, E; Cecchele, A; Morena, F; Sanvito, F; Matafora, V; Cattaneo, A; Della Volpe, L; Gnani, D; Paulis, M; Susani, L; Martino, S; Di Micco, R; Bachi, A; Gritti, A
Dissecting the Transcriptional and Epigenetic Landscape of hiPSC-Derived Neural Stem and Progenitor Cells: Implications for Cell Therapy Approaches
2021-01-01 Meneghini, V; Luciani, M; Garsia, C; Petiti, L; Cifola, I; Beretta, S; Merelli, I; Peano, C; Miccio, A; Gritti, A
Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes
2021-01-01 Mangiameli, E; Cecchele, A; Morena, F; Sanvito, F; Matafora, V; Cattaneo, A.; della Volpe, L; Gnani, D; Paulis, M; Susani, L; Martino, S; Di Micco, R; Bachi, A; Gritti, A.
Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System
2021-01-01 Meneghini, Vasco; Peviani, Marco; Luciani, Marco; Zambonini, Giada; Gritti, Angela
| Titolo | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|
| Perspective on innovative therapies for globoid cell leukodystrophy | 1-gen-2016 | Ricca, A.; Gritti, A. | |
| Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy | 1-gen-2016 | Meneghini, Vasco; Lattanzi, Annalisa; Tiradani, Luigi; Bravo, Gabriele; Morena, Francesco; Sanvito, Francesca; Calabria, Andrea; Bringas, John; Fisher-Perkins, Jeanne M; Dufour, Jason P; Baker, Kate C; Doglioni, Claudio; Montini, Eugenio; Bunnell, Bruce A; Bankiewicz, Krystof; Martino, Sabata; Naldini, Luigi; Gritti, Angela | |
| Early sulfatide storage and progressive neuronal and glial cell dysfunction revealed by human iPSC-based models of metachromatic leukodystrophy are rescued by lentiviral-mediated restoration of ARSA activity | 1-gen-2017 | Frati, ; G, ; Luciani, ; M, ; Meneghini, ; V, ; De, Cicco; S, ; Stahlman, ; M, ; Grossi, ; S, ; Morena, ; F, ; Blomqvist, ; M, ; Filocamo, ; M, ; Martino, ; S, ; Gritti, A | |
| Genome-Wide Definition of Regulatory Regions and Transcripts During the Transition from Pluripotent to Neural-Restricted Stem Cells | 1-gen-2017 | Meneghini, ; V, ; Luciani, ; M, ; Petiti, ; L, ; Cifola, ; I, ; Peano, ; C, ; Gritti, A; Miccio, A | |
| Neuronal and glial cells derived from a murine model of GM2-gangliosidosis display typical hallmarks of pathology and represent a valuable model to test gene therapy strategies | 1-gen-2017 | Ornaghi, ; F, ; Tedeschi, ; F, ; Sala, ; D, ; Ricca, ; A, ; Di, Tomaso; T, ; Morena, ; F, ; Martino, ; S, ; Gritti, A | |
| Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy | 1-gen-2017 | Meneghini, Vasco; Frati, Giacomo; Sala, Davide; De Cicco, Silvia; Luciani, Marco; Cavazzin, Chiara; Paulis, Marianna; Mentzen, Wieslawa; Morena, Francesco; Giannelli, Serena; Sanvito, Francesca; Villa, Anna; Bulfone, Alessandro; Broccoli, Vania; Martino, Sabata; Gritti, Angela | |
| Two factor-based reprogramming of rodent and human fibroblasts into Schwann cells | 1-gen-2017 | Mazzara, P. G.; Massimino, L; Pellegatta, M; Ronchi, G; Ricca, A; Iannielli, A; Giannelli, S. G.; Cursi, M; Cancellieri, C; Sessa, A; Del Carro, U; Quattrini, A; Geuna, S; Gritti, A; Taveggia, C; Broccoli, V. | |
| Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy | 1-gen-2018 | Frati, Giacomo; Luciani, Marco; Meneghini, Vasco; De Cicco, Silvia; Ståhlman, Marcus; Blomqvist, Maria; Grossi, Serena; Filocamo, Mirella; Morena, Francesco; Menegon, Andrea; Martino, Sabata; Gritti, Angela | |
| Optimized bicistronic lentiviral vectors to correct b-hexosaminidase deficiency in neural and hematopoietic stem/progenitor cells and progeny | 1-gen-2019 | Ornaghi, F; Sala, D; Tedeschi, F; Bazzucchi, M; Morena, F; Martino, S; Gritti, A | |
| Integrated computational analysis highlights unique miRNA signatures in the subventricular zone and striatum of GM2 gangliosidosis animal models | 1-gen-2019 | Morena, F; Oikonomou, V; Argentati, C; Bazzucchi, M; Emiliani, C; Gritti, A; Martino, S. | |
| Genome-wide analyses of regulatory regions and transcripts in hiPSC-derived neural stem/progenitor cells to define their safety and efficacy in cell therapy approaches for neurological disorders | 1-gen-2019 | Meneghini, V; Luciani, M; Petiti, L; Cifola, I; Peano, C; Miccio, A; Gritti, A | |
| Exploiting targeted epigenetic editing to increase the yield, homogeneity and purity of human iPSCderived oligodendrocyte cell populations | 1-gen-2019 | Luciani, M; Ciccarelli, E; Migliara, A; Lombardo, A; Gritti, A | |
| In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion | 1-gen-2020 | Ricca, A; Cascino, F; Morena, F; Martino, S; Gritti, A. | |
| Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis | 1-gen-2020 | Ornaghi, F; Sala, D; Tedeschi, F; Maffia, M. C.; Bazzucchi, M; Morena, F; Valsecchi, M; Aureli, M; Martino, S; Gritti, A. | |
| Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases | 1-gen-2020 | Luciani, Marco; Gritti, Angela; Meneghini, Vasco | |
| Chimeric Lysosomal Enzymes with Improved Bioavailability to Advance Gene Therapy Strategies for Globoid Cell Leukodystrophy | 1-gen-2021 | Cascino, F; Ricca, A; Morena, F; Martino, S; Gritti, A | |
| A Human IPSC-Based Model of Globoid Cell Leukodystrophy Uncovers Early Neurodevelopmental Defects | 1-gen-2021 | Mangiameli, E; Cecchele, A; Morena, F; Sanvito, F; Matafora, V; Cattaneo, A; Della Volpe, L; Gnani, D; Paulis, M; Susani, L; Martino, S; Di Micco, R; Bachi, A; Gritti, A | |
| Dissecting the Transcriptional and Epigenetic Landscape of hiPSC-Derived Neural Stem and Progenitor Cells: Implications for Cell Therapy Approaches | 1-gen-2021 | Meneghini, V; Luciani, M; Garsia, C; Petiti, L; Cifola, I; Beretta, S; Merelli, I; Peano, C; Miccio, A; Gritti, A | |
| Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes | 1-gen-2021 | Mangiameli, E; Cecchele, A; Morena, F; Sanvito, F; Matafora, V; Cattaneo, A.; della Volpe, L; Gnani, D; Paulis, M; Susani, L; Martino, S; Di Micco, R; Bachi, A; Gritti, A. | |
| Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System | 1-gen-2021 | Meneghini, Vasco; Peviani, Marco; Luciani, Marco; Zambonini, Giada; Gritti, Angela |
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