AMENDOLA, MARIO
AMENDOLA, MARIO
Dipartimento di Medicina Clinica e Sperimentale
"Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof"
2003-01-01 Naldini, L; Amendola, M; Vigna, E
A combination of cyclophosphamide, interleukin-2 allow CD4+ T cells converted to Tregs to control scurfy syndrome.
2021-01-01 8., Delville M; Bellier, F; Leon, J; Klifa, R; Lizot, S; Vinçon, H; Sobrino, S; Thouenon, R; Marchal, A; Garrigue, A; Olivre, J; Charbonnier, S; Lagresle-Peyrou, C; Amendola, M; Schambach, A; Gross, Da; Lamarthée, B; Benoist, C; Zuber, J; André, I; Cavazzana, M; Six, E
A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector)
2013-01-01 Amendola, M; Giustacchini, A; Gentner, B; Naldini, L.
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
2022-01-01 Antoniou, Panagiotis; Hardouin, Giulia; Martinucci, Pierre; Frati, Giacomo; Felix, Tristan; Chalumeau, Anne; Fontana, Letizia; Martin, Jeanne; Masson, Cecile; Brusson, Megane; Maule, Giulia; Rosello, Marion; Giovannangeli, Carine; Abramowski, Vincent; de Villartay, Jean-Pierre; Concordet, Jean-Paul; Del Bene, Filippo; El Nemer, Wassim; Amendola, Mario; Cavazzana, Marina; Cereseto, Anna; Romano, Oriana; Miccio, Annarita
Bifunctional lentiviral vectors allowing the bs-globin silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of sickle cell disease
2020-01-01 M, Brusson; F, Mavilio; M, Cavazzana; Amendola, M; A, Miccio
Bifunctional vectors allowing Bcl11A silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of B-hemoglobinopathies
2019-01-01 Brusson, M; Mavilio, F; Cavazzana, M; Amendola, M; 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.
Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters
2005-01-01 Amendola, M; Venneri, Ma; Biffi, A; Vigna, E; Naldini, L.
Correction of beta-thalassemia phenotype by genetically engineered hematopoietic stem cells
2019-01-01 Amendola, M; Pavani, G
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
CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy
2022-01-01 Amendola, M; Brusson, M; Miccio, A.
Easy quantitative assessment of genome editing by sequence trace decomposition
2014-01-01 Brinkman, Ek; Chen, T; Amendola, M; van Steensel, B
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.
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.
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.
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.
Enhancing utrophin expression in the cell by inducing mutations within the regulatory elements and therapeutics use thereof
2020-01-01 Amendola, M; S, Guiraud
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
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.
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.
Titolo | Data di pubblicazione | Autore(i) | File |
---|---|---|---|
"Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof" | 1-gen-2003 | Naldini, L; Amendola, M; Vigna, E | |
A combination of cyclophosphamide, interleukin-2 allow CD4+ T cells converted to Tregs to control scurfy syndrome. | 1-gen-2021 | 8., Delville M; Bellier, F; Leon, J; Klifa, R; Lizot, S; Vinçon, H; Sobrino, S; Thouenon, R; Marchal, A; Garrigue, A; Olivre, J; Charbonnier, S; Lagresle-Peyrou, C; Amendola, M; Schambach, A; Gross, Da; Lamarthée, B; Benoist, C; Zuber, J; André, I; Cavazzana, M; Six, E | |
A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector) | 1-gen-2013 | Amendola, M; Giustacchini, A; Gentner, B; Naldini, L. | |
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression | 1-gen-2022 | Antoniou, Panagiotis; Hardouin, Giulia; Martinucci, Pierre; Frati, Giacomo; Felix, Tristan; Chalumeau, Anne; Fontana, Letizia; Martin, Jeanne; Masson, Cecile; Brusson, Megane; Maule, Giulia; Rosello, Marion; Giovannangeli, Carine; Abramowski, Vincent; de Villartay, Jean-Pierre; Concordet, Jean-Paul; Del Bene, Filippo; El Nemer, Wassim; Amendola, Mario; Cavazzana, Marina; Cereseto, Anna; Romano, Oriana; Miccio, Annarita | |
Bifunctional lentiviral vectors allowing the bs-globin silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of sickle cell disease | 1-gen-2020 | M, Brusson; F, Mavilio; M, Cavazzana; Amendola, M; A, Miccio | |
Bifunctional vectors allowing Bcl11A silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of B-hemoglobinopathies | 1-gen-2019 | Brusson, M; Mavilio, F; Cavazzana, M; Amendola, M; 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. | |
Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters | 1-gen-2005 | Amendola, M; Venneri, Ma; Biffi, A; Vigna, E; Naldini, L. | |
Correction of beta-thalassemia phenotype by genetically engineered hematopoietic stem cells | 1-gen-2019 | Amendola, M; Pavani, G | |
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 | |
CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy | 1-gen-2022 | Amendola, M; Brusson, M; Miccio, A. | |
Easy quantitative assessment of genome editing by sequence trace decomposition | 1-gen-2014 | Brinkman, Ek; Chen, T; Amendola, M; van Steensel, B | |
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. | |
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. | |
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. | |
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. | |
Enhancing utrophin expression in the cell by inducing mutations within the regulatory elements and therapeutics use thereof | 1-gen-2020 | Amendola, M; S, Guiraud | |
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 | |
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. | |
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. |