IRIS

AMENDOLA, MARIO

AMENDOLA, MARIO  

Dipartimento di Medicina Clinica e Sperimentale  

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Risultati 1 - 20 di 48 (tempo di esecuzione: 0.043 secondi).
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.
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease 1-gen-2025 Chalumeau, Anne; Bou Dames, Maria; Fontana, Letizia; Amistadi, Simone; Antoniou, Panagiotis; Loganathan, Priyanka; Mombled, Margaux; Corre, Guillaume; Peterka, Martin; Amendola, Mario; Giovannangeli, Carine; Maresca, Marcello; Miccio, Annarita; Brusson, Mégane
Advanced Imaging and Cytometric Techniques to Characterize Lipid Accumulation in Wolman Disease 1-gen-2025 Laurent, Marine; Cosette, Jérémie; Pavani, Giulia; Bayol, Sarah; Jenny, Christine; Harb, Rim; Oustelandt, Julie; Brassier, Anais; Stockholm, Daniel; Amendola, Mario
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