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Annarita Miccio, PhD
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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

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Elisa Magrin, PhD, Michaela Semeraro, MD PhD, Alessandra Magnani, MD PhD, Hervé Puy, MD PhD, Annarita Miccio, PhD, Nicolas Hebert, Jean-Sebastien Diana, MD, Francois Lefrere, MD, Felipe Suarez, MD PhD, Olivier Hermine, MD PhD, Valentine Brousse, MD, Catherine Poirot, MD PhD, Philippe Bourget, PharmD, PhD, Wassim El Nemer, PhD, Isabelle Guichard, MD, Despina Moshous, MD PhD, Benedicte Neven, MD, Fabrice Monpoux, MD, Marilyne Poirée, MD, Pablo Bartolucci, MD PhD, Jean-François Meritet, MD, David Grévent, MD, Thibaud Lefebvre, Pharm D.,PhD, Mohammed Asmal, MD PhD, Erin Whitney, MBA, Marisa Gayron, MS, Wenmei Huang, PhD MS, Isabelle Funck-Brentano, PsyD, Mariane de Montalembert, MD PhD, Laure Joseph, MD, Jean-Antoine Ribeil, MD PhD, Marina Cavazzana
Journal: Blood
Blood (2019) 134 (Supplement_1): 3358.
https://doi.org/10.1182/blood-2019-127393
Published: 2019
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

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

Nicolas Hebert, Elisa Magrin, PhD, Annarita Miccio, PhD, Kiger Laurent, PhD, Nguyen-Peyre Kim-Anh, PhD, Laure Joseph, MD, Alessandra Magnani, MD PhD, Chloé Couzin, MD, Wassim El Nemer, PhD, France Pirenne, MD PhD, Olivier Negre, PhD, Jean-Antoine Ribeil, MD PhD, Isabelle André-Schmutz, PhD, Pablo Bartolucci, MD PhD, Marina Cavazzana, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2195.
https://doi.org/10.1182/blood-2018-99-115973
Published: 2018
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
Journal Articles

Re-Creating Hereditary Persistence of Fetal Hemoglobin (HPFH) to Treat Sickle Cell Disease (SCD) and β-Thalassemia

Michelle I Lin, PhD, Elizabeth J Paik, PhD, Bibhu P Mishra, PhD, Song Chou, PhD, Ying Zhang, PhD, Kaleigh Tomkinson, MS, Michael A Pettiglio, BS, Andrew Sanginario, BS, Amanda Woo, BS, Min Jin Lee, BS, Melanie Allen, MS, Thomas J Cradick, PhD, Tirtha Chakraborty, PhD, Matthew Porteus, MD PhD, Ciaran M Lee, PhD, Gang Bao, PhD, Annarita Miccio, PhD, Annalisa Lattanzi, PhD, Fulvio Mavilio, PhD, Chad A Cowan, PhD, Rodger Novak, MD, Ante S Lundberg, MD
Journal: Blood
Blood (2016) 128 (22): 4708.
https://doi.org/10.1182/blood.V128.22.4708.4708
Published: 2016
Journal Articles

Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies

Ciaran Lee, PhD, Gang Bao, PhD, Matthew H. Porteus, MD PhD, Tatjana Cornu, PhD, Annarita Miccio, PhD, Thomas Cradick, Toni Cathomen, PhD, Ante Lundberg, MD, Fulvio Mavilio, PhD
Journal: Blood
Blood (2015) 126 (23): 3376.
https://doi.org/10.1182/blood.V126.23.3376.3376
Published: 2015
Journal Articles

FOG-1 Requires NuRD to Promote Hematopoiesis and Maintain Lineage Fidelity within the Megakaryocytic–Erythroid Compartment.

Gregory D Gregory, PhD, Annarita Miccio, PhD, Alexey Bersenev, MD, Yuhuan Wang, PhD, Wei Hong, MD, Zhe Zhang, Mortimer Poncz, MD, Wei Tong, PhD, Gerd A Blobel, MD, PhD
Journal: Blood
Blood (2009) 114 (22): 702.
https://doi.org/10.1182/blood.V114.22.702.702
Published: 2009