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Giacomo Frati
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Journal Articles

Targeted Base Editing Strategies for Beta-Hemoglobinopathies

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Marta Zoccheddu, Anne Chalumeau, Giacomo Frati, Sandra Manceau, Laure Joseph, Oriana Romano, Marina Cavazzana, Annarita Miccio
Journal: Blood
Blood (2022) 140 (Supplement 1): 7778–7779.
https://doi.org/10.1182/blood-2022-162362
Published: 2022
Journal Articles

Base Editing-Mediated Dissection of the -200 Region of the γ-Globin Promoters to Induce Fetal Hemoglobin and Rescue Sickle Cell Disease and β-Thalassemia

Panagiotis Antoniou, Giulia Hardouin, Pierre Martinucci, Tristan Felix, Letizia Fontana, Jeanne Martin, Giacomo Frati, Megane Brusson, Marion Rosello, Giulia Maule, Filippo Del Bene, Anna Cereseto, Wassim El Nemer, Jean-Paul Concordet, Annarita Miccio
Journal: Blood
Blood (2021) 138 (Supplement 1): 562.
https://doi.org/10.1182/blood-2021-149710
Published: 2021
Journal Articles

Pre-Clinical Development of a Highly Efficient TALEN ® -Based Correction of the β-Globin Gene in Patient-Derived Hematopoietic Stem and Progenitor Cells (HSPCs) to Treat Sickle Cell Disease

Arianna Moiani, Patrick Hong, Gil Letort, Sabrina Lizot, Alexa Chirinos, Sonal Temburni-Blake, Sara Nik, Louisa Mayer, Sophie Leduc, Noemie Pinard, Chloe Foray, Alex Boyne, Selena Kazancioglu, Giacomo Frati, Tristan Felix, Anne Chalumeau, Annarita Miccio, Mathilde Dusseaux, Agnès Gouble, Mark G. Frattini, Carrie Brownstein, Aymeric Duclert, Cecile Shiffer-Mannoui, Alexandre Juillerat, Philippe Duchateau, Julien Valton
Journal: Blood
Blood (2021) 138 (Supplement 1): 1856.
https://doi.org/10.1182/blood-2021-153549
Published: 2021
Journal Articles

Fetal Hemoglobin Rescues Ineffective Erythropoiesis in Sickle Cell Disease

Sara El Hoss, PhD, Sylvie Cochet, Auria Godard, MS, Hongxia Yan, MS, Michaël Dussiot, PhD, Giacomo Frati, PhD, Bénédicte Boutonnat-Faucher, MD, Sandrine Laurance, PhD, Olivier Renaud, PhD, Laure Joseph, MD, Annarita Miccio, PhD, Valentine Brousse, MDPhD, Mohandas Narla, DSc, Wassim El Nemer, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 14–15.
https://doi.org/10.1182/blood-2020-137477
Published: 2020
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

Bone Marrow-Derived Cells Regenerate Kit+ Cardiac Stem Cells (CSCs) in Damaged Heart.

Francesco Cerisoli, Lucio Barile, Roberto Gaetani, Letizia Cassinelli, Giacomo Frati, Alessandro Giacomello, Elisa Messina, Sergio Ottolenghi, Maria Cristina Magli
Journal: Blood
Blood (2006) 108 (11): 1681.
https://doi.org/10.1182/blood.V108.11.1681.1681
Published: 2006