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Journal Articles

Vexas Syndrome Is a New Cause of p53-Mediated Erythroblastopenia

François Rodrigues, Giulia Hardouin, Sara El Hoss, Aya Ghoul, Emilie-Fleur Gautier, Michael Dussiot, Sandy Peltier, Pascal Amireault, Annarita Miccio, Vincent Jachiet, Thiago Trovati Maciel, Sophie Georgin Lavialle, Julien Rossignol, Arsène Mekhinian, Olivier Kosmider, Manuel Rodriguez, Olivier Hermine

Blood (2024) 144 (Supplement 1): 36.

https://doi.org/10.1182/blood-2024-208122

Published: 2024

Journal Articles

Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cécile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio

Blood (2023) 141 (10): 1169–1179.

https://doi.org/10.1182/blood.2022016629

Published: 2023

Includes: Supplemental data

Journal Articles

Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing

Panagiotis Antoniou, Mathieu von Joest, Letizia Fontana, Giulia Hardouin, Pierre Martinucci, Marina Cavazzana, Annarita Miccio

Blood (2022) 140 (Supplement 1): 7788–7789.

https://doi.org/10.1182/blood-2022-166330

Published: 2022

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

Blood (2022) 140 (Supplement 1): 7778–7779.

https://doi.org/10.1182/blood-2022-162362

Published: 2022

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

Blood (2021) 138 (Supplement 1): 3973.

https://doi.org/10.1182/blood-2021-151076

Published: 2021

Journal Articles

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

Blood (2021) 138 (Supplement 1): 1854.

https://doi.org/10.1182/blood-2021-152331

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

Blood (2021) 138 (Supplement 1): 1856.

https://doi.org/10.1182/blood-2021-153549

Published: 2021

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

Blood (2021) 138 (Supplement 1): 562.

https://doi.org/10.1182/blood-2021-149710

Published: 2021

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

Blood (2020) 136 (Supplement 1): 33.

https://doi.org/10.1182/blood-2020-139107

Published: 2020

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

Blood (2020) 136 (Supplement 1): 14–15.

https://doi.org/10.1182/blood-2020-137477

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

Blood (2019) 134 (Supplement_1): 4635.

https://doi.org/10.1182/blood-2019-123192

Published: 2019

Journal Articles

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

Blood (2019) 134 (Supplement_1): 3358.

https://doi.org/10.1182/blood-2019-127393

Published: 2019

Journal Articles

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

Elisa Magrin, Annarita Miccio, Marina Cavazzana

Blood (2019) 134 (15): 1203–1213.

https://doi.org/10.1182/blood.2019000949

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

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 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

Blood (2018) 131 (17): 1960–1973.

https://doi.org/10.1182/blood-2017-10-811505

Published: 2018

Includes: Supplemental data

Journal Articles

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

Blood (2016) 128 (22): 4708.

https://doi.org/10.1182/blood.V128.22.4708.4708

Published: 2016

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

Blood (2016) 128 (22): 321.

https://doi.org/10.1182/blood.V128.22.321.321

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

Blood (2015) 126 (23): 3376.

https://doi.org/10.1182/blood.V126.23.3376.3376

Published: 2015

Journal Articles

FOG1 requires NuRD to promote hematopoiesis and maintain lineage fidelity within the megakaryocytic-erythroid compartment

Gregory D. Gregory, Annarita Miccio, Alexey Bersenev, Yuhuan Wang, Wei Hong, Zhe Zhang, Mortimer Poncz, Wei Tong, Gerd A. Blobel

Blood (2010) 115 (11): 2156–2166.

https://doi.org/10.1182/blood-2009-10-251280

Published: 2010

Includes: Supplemental data

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

Blood (2009) 114 (22): 702.

https://doi.org/10.1182/blood.V114.22.702.702

Published: 2009

NARROW

Vexas Syndrome Is a New Cause of p53-Mediated Erythroblastopenia

Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing

Targeted Base Editing Strategies for Beta-Hemoglobinopathies

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

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

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

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

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

Fetal Hemoglobin Rescues Ineffective Erythropoiesis in Sickle Cell Disease

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

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

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

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

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

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

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

Gene Editing with Crispr-Cas9 for Treating Beta-Hemoglobinopathies

FOG1 requires NuRD to promote hematopoiesis and maintain lineage fidelity within the megakaryocytic-erythroid compartment

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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