Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease
2020-01-01 12., Weber L; Frati, G; Felix, T; Hardouin, G; Casini, A; Wollenschlaeger, C; Meneghini, V; Masson, C; De Cian, A; Chalumeau, A; Mavilio, F; Amendola, M; Andre-Schmutz, I; Cereseto, A; El Nemer, W; Concordet, Jp; Giovannangeli, C; Cavazzana, M; Miccio, A.
Mechanisms and dynamics of nuclear lamina–genome interactions
2014-01-01 Amendola, M; van Steensel, B
Effects of phosphorylation and neuronal activity on the control of synapse formation by synapsin I
2011-01-01 Perlini, Le; Botti, F; Fornasiero, Ef; Giannandrea, M; Bonanomi, D; Amendola, M; Naldini, L; Benfenati, F; Valtorta, F.
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
2021-01-01 7., Pavani G; Fabiano, A; Laurent, M; Amor, F; Cantelli, E; Chalumeau, A; Concordet, Jp; Mavilio, F; Ferrari, G.; Miccio, A; Amendola, M
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
2007-01-01 Brown, Bd; Gentner, B; Cantore, A; Colleoni, S; Amendola, M; Zingale, A; Baccarini, A; Lazzari, G; Galli, C; Naldini, L.
Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective
2021-01-01 Amendola, M; Bedel, A; Buj-Bello, A; Carrara, M; Concordet, Jp; Frati, G; Gilot, D; Giovannangeli, C; Gutierrez-Guerrero, A; Laurent, M; Miccio, A; Moreau-Gaudry, F; Sourd, C; Valton, J; Verhoeyen, E.
Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3
2008-01-01 Allan, Se; Alstad, An; Merindol, N; Crellin, Nk; Amendola, M; Bacchetta, R; Naldini, L; Roncarolo, Mg; Soudeyns, H; Levings, Mk.
Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke
2009-01-01 Carlén, M; Meletis, K; Göritz, C; Darsalia, V; Evergren, E; Tanigaki, K; Amendola, M; Barnabé-Heider, F; Yeung, Ms; Naldini, L; Honjo, T; Kokaia, Z; Shupliakov, O; Cassidy, Rm; Lindvall, O; Frisén, J.
Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors
2009-01-01 Bobisse, S; Rondina, M; Merlo, A; Tisato, V; Mandruzzato, S; Amendola, M; Naldini, L; Willemsen, Ra; Debets, R; Zanovello, P; Rosato, A.
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
2020-01-01 Pavani, G; Laurent, M; Fabiano, A; Cantelli, E; Sakkal, A; Corre, G; Lenting, Pj; Concordet, Jp; Toueille, M; Miccio, A; Amendola, M
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
2018-01-01 Antoniani, C; Meneghini, V; Lattanzi, A; Felix, T; Romano, O; Magrin, E; Weber, L; Pavani, G; El Hoss, S; Kurita, R; Nakamura, Y; Cradick, Tj; Lundberg, As; Porteus, M; Amendola, M; El Nemer, W; Cavazzana, M; Mavilio, F; Miccio, A
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
2022-01-01 5., Ramadier S; Chalumeau, A; Felix, T; Othman, N; Aknoun, S; Casini, A; Maule, G; Masson, C; De Cian, A; Frati, G; Brusson, M; Concordet, Jp; Cavazzana, M; Cereseto, A; El Nemer, W; Amendola, M; Wattellier, B; Meneghini, V; Miccio, A.
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
2010-01-01 Gentner, B; Visigalli, I; Hiramatsu, H; Lechman, E; Ungari, S; Giustacchini, A; Schira, G; Amendola, M; Quattrini, A; Martino, S; Orlacchio, A; Dick, Je; Biffi, A; Naldini, L.
Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability
2011-01-01 Passerini, L; Olek, S; Di Nunzio, S; Barzaghi, F; Hambleton, S; Abinun, M; Tommasini, A; Vignola, S; Cipolli, M; Amendola, M; Naldini, L; Guidi, L; Cecconi, M; Roncarolo, Mg; Bacchetta, R.
Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector
2005-01-01 Vigna, E; Amendola, M; Benedicenti, F; Simmons, Ad; Follenzi, A; Naldini, L.
Stable knockdown of microRNA in vivo by lentiviral vectors.
2009-01-01 Gentner, B; Schira, G; Giustacchini, A; Amendola, M; Brown, Bd; Ponzoni, M; Naldini, L.
Easy quantitative assessment of genome editing by sequence trace decomposition
2014-01-01 Brinkman, Ek; Chen, T; Amendola, M; van Steensel, B
GATA factor-mediated gene regulation in human erythropoiesis
2020-01-01 Romano, O; Petiti, L; Felix, T; Meneghini, V; Portafax, M; Antoniani, C; Amendola, M; Bicciato, S; Peano, C; Miccio, A
The cohesin release factor WAPL restricts chromatin loop extension
2017-01-01 Haarhuis, Jht; van der Weide, Rh; Blomen, Va; Yáñez-Cuna, Jo; Amendola, M; van Ruiten, Ms; Krijger, Phl; Teunissen, H; van Ruiten, Ms; Medema, Rh; van Steensel, B; Brummelkamp, Tr; de Wit, E; Rowland, Bd
Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models
2010-01-01 Lattanzi, A; Neri, M; Maderna, C; di Girolamo, I; Martino, S; Orlacchio, A; Amendola, M; Naldini, L; Gritti, A.
| Titolo | Data di pubblicazione | Autore(i) | File |
|---|---|---|---|
| Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease | 1-gen-2020 | 12., Weber L; Frati, G; Felix, T; Hardouin, G; Casini, A; Wollenschlaeger, C; Meneghini, V; Masson, C; De Cian, A; Chalumeau, A; Mavilio, F; Amendola, M; Andre-Schmutz, I; Cereseto, A; El Nemer, W; Concordet, Jp; Giovannangeli, C; Cavazzana, M; Miccio, A. | |
| Mechanisms and dynamics of nuclear lamina–genome interactions | 1-gen-2014 | Amendola, M; van Steensel, B | |
| Effects of phosphorylation and neuronal activity on the control of synapse formation by synapsin I | 1-gen-2011 | Perlini, Le; Botti, F; Fornasiero, Ef; Giannandrea, M; Bonanomi, D; Amendola, M; Naldini, L; Benfenati, F; Valtorta, F. | |
| Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells | 1-gen-2021 | 7., Pavani G; Fabiano, A; Laurent, M; Amor, F; Cantelli, E; Chalumeau, A; Concordet, Jp; Mavilio, F; Ferrari, G.; Miccio, A; Amendola, M | |
| Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state | 1-gen-2007 | Brown, Bd; Gentner, B; Cantore, A; Colleoni, S; Amendola, M; Zingale, A; Baccarini, A; Lazzari, G; Galli, C; Naldini, L. | |
| Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective | 1-gen-2021 | Amendola, M; Bedel, A; Buj-Bello, A; Carrara, M; Concordet, Jp; Frati, G; Gilot, D; Giovannangeli, C; Gutierrez-Guerrero, A; Laurent, M; Miccio, A; Moreau-Gaudry, F; Sourd, C; Valton, J; Verhoeyen, E. | |
| Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3 | 1-gen-2008 | Allan, Se; Alstad, An; Merindol, N; Crellin, Nk; Amendola, M; Bacchetta, R; Naldini, L; Roncarolo, Mg; Soudeyns, H; Levings, Mk. | |
| Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke | 1-gen-2009 | Carlén, M; Meletis, K; Göritz, C; Darsalia, V; Evergren, E; Tanigaki, K; Amendola, M; Barnabé-Heider, F; Yeung, Ms; Naldini, L; Honjo, T; Kokaia, Z; Shupliakov, O; Cassidy, Rm; Lindvall, O; Frisén, J. | |
| Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors | 1-gen-2009 | Bobisse, S; Rondina, M; Merlo, A; Tisato, V; Mandruzzato, S; Amendola, M; Naldini, L; Willemsen, Ra; Debets, R; Zanovello, P; Rosato, A. | |
| Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins | 1-gen-2020 | Pavani, G; Laurent, M; Fabiano, A; Cantelli, E; Sakkal, A; Corre, G; Lenting, Pj; Concordet, Jp; Toueille, M; Miccio, A; Amendola, M | |
| Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus | 1-gen-2018 | Antoniani, C; Meneghini, V; Lattanzi, A; Felix, T; Romano, O; Magrin, E; Weber, L; Pavani, G; El Hoss, S; Kurita, R; Nakamura, Y; Cradick, Tj; Lundberg, As; Porteus, M; Amendola, M; El Nemer, W; Cavazzana, M; Mavilio, F; Miccio, A | |
| Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease | 1-gen-2022 | 5., Ramadier S; Chalumeau, A; Felix, T; Othman, N; Aknoun, S; Casini, A; Maule, G; Masson, C; De Cian, A; Frati, G; Brusson, M; Concordet, Jp; Cavazzana, M; Cereseto, A; El Nemer, W; Amendola, M; Wattellier, B; Meneghini, V; Miccio, A. | |
| Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy | 1-gen-2010 | Gentner, B; Visigalli, I; Hiramatsu, H; Lechman, E; Ungari, S; Giustacchini, A; Schira, G; Amendola, M; Quattrini, A; Martino, S; Orlacchio, A; Dick, Je; Biffi, A; Naldini, L. | |
| Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability | 1-gen-2011 | Passerini, L; Olek, S; Di Nunzio, S; Barzaghi, F; Hambleton, S; Abinun, M; Tommasini, A; Vignola, S; Cipolli, M; Amendola, M; Naldini, L; Guidi, L; Cecconi, M; Roncarolo, Mg; Bacchetta, R. | |
| Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector | 1-gen-2005 | Vigna, E; Amendola, M; Benedicenti, F; Simmons, Ad; Follenzi, A; Naldini, L. | |
| Stable knockdown of microRNA in vivo by lentiviral vectors. | 1-gen-2009 | Gentner, B; Schira, G; Giustacchini, A; Amendola, M; Brown, Bd; Ponzoni, M; Naldini, L. | |
| Easy quantitative assessment of genome editing by sequence trace decomposition | 1-gen-2014 | Brinkman, Ek; Chen, T; Amendola, M; van Steensel, B | |
| GATA factor-mediated gene regulation in human erythropoiesis | 1-gen-2020 | Romano, O; Petiti, L; Felix, T; Meneghini, V; Portafax, M; Antoniani, C; Amendola, M; Bicciato, S; Peano, C; Miccio, A | |
| The cohesin release factor WAPL restricts chromatin loop extension | 1-gen-2017 | Haarhuis, Jht; van der Weide, Rh; Blomen, Va; Yáñez-Cuna, Jo; Amendola, M; van Ruiten, Ms; Krijger, Phl; Teunissen, H; van Ruiten, Ms; Medema, Rh; van Steensel, B; Brummelkamp, Tr; de Wit, E; Rowland, Bd | |
| Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models | 1-gen-2010 | Lattanzi, A; Neri, M; Maderna, C; di Girolamo, I; Martino, S; Orlacchio, A; Amendola, M; Naldini, L; Gritti, A. |
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