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Mario Amendola
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Journal Articles

Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies

Megane Brusson, Anne Chalumeau, Pierre Martinucci, Valentina Poletti, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio
Journal: Blood
Blood (2021) 138 (Supplement 1): 3973.
https://doi.org/10.1182/blood-2021-151076
Published: 2021
Journal Articles

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

Marianne Delville, Florence Bellier, Juliette Leon, Roman Klifa, Sabrina Lizot, Hélène Vinçon, Steicy Sobrino, Romane Thouenon, Armance Marchal, Alexandrine Garrigue, Juliette Olivré, Soëli Charbonnier, Chantal Lagresle-Peyrou, Mario Amendola, Axel Schambach, David Gross, Baptiste Lamarthée, Christophe Benoist, Julien Zuber, Isabelle André, Marina Cavazzana, Emmanuelle Six
Journal: Blood
Blood (2021) 137 (17): 2326–2336.
https://doi.org/10.1182/blood.2020009187
Published: 2021
Includes: Supplemental data
Journal Articles

Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies

Giacomo Frati, PhD, Panagiotis Antoniou, MSc, Giulia Hardouin, Bochra Mlaya, MSc, Leslie Weber, Tristan Felix, Antonio Casini, Marion Rosello, Anne Chalumeau, Mario Amendola, Adrian J. Thrasher, MBBS, PhD FMedSci, Giandomenico Turchiano, PhD, Filippo Del Bene, Wassim El Nemer, PhD, Anna Cereseto, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, MD PhD, Annarita Miccio, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 33.
https://doi.org/10.1182/blood-2020-139107
Published: 2020
Journal Articles

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

Leslie Weber, Giacomo Frati, Tristan Felix, Clara Wollenschlaeger, Antonio Casini, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, PhD, Mario Amendola, Isabelle André, PhD, Anna Cereseto, Wassim El Nemer, PhD, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4635.
https://doi.org/10.1182/blood-2019-123192
Published: 2019
Journal Articles

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Journal: Blood
Blood (2018) 131 (17): 1960–1973.
https://doi.org/10.1182/blood-2017-10-811505
Published: 2018
Includes: Supplemental data
Journal Articles

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

Chiara Antoniani, MSc, Vasco Meneghini, Annalisa Lattanzi, PhD, Giulia Pavani, PhD, Tristan Felix, MSc, Fatima Amor, MSc, Oriana Romano, Elisa Magrin, PhD, Leslie Weber, MSc, Thomas Cradick, Ante S Lundberg, MD, Matthew Porteus, MD PhD, Mario Amendola, Marina Cavazzana, MD PhD, Fulvio Mavilio, PhD, Annarita Miccio, PhD
Journal: Blood
Blood (2016) 128 (22): 321.
https://doi.org/10.1182/blood.V128.22.321.321
Published: 2016