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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

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Janet L. Kwiatkowski, MD MSCE, Mark C. Walters, MD, Suradej Hongeng, MD, Franco Locatelli, MD PhD, John E.J. Rasko, BSc, MBBS, PhD, Marina Cavazzana, MD PhD, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 51–52.

https://doi.org/10.1182/blood-2020-135850

Published: 2020

Journal Articles

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Jean-Sebastien Diana, MD, Sandra Manceau, PhD, Thierry Leblanc, MD, Chloé Couzin, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Matthieux Bendavid, MD, Laure Joseph, MD, Marianne Delville, MD, Stephane Blanche, MD, Jean Soulier, MD PhD, Marina Cavazzana, MD PhD, Francois Lefrere, MD

Blood (2019) 134 (Supplement_1): 1970.

https://doi.org/10.1182/blood-2019-128051

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Chantal Lagresle-Peyrou, PhD, Aurélien Olichon, Hanem Sadek, Philippe Roche, Claudine Tardy, Cindy Da Silva, Alexandrine Garrigue, Alain Fischer, MD PhD, Despina Moshous, MD PhD, Yves Collette, Capucine Picard, MD PhD, Jean-Laurent Casanova, PhD MD, Isabelle André, PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 3742.

https://doi.org/10.1182/blood-2019-126855

Published: 2019

Journal Articles

Therapeutic Plasma Exchange in Pediatrics for Immunologic Disorders; Tolerated and Safe Process for Pediatric Life-Threatening Conditions

Blood (2019) 134 (Supplement_1): 4986.

https://doi.org/10.1182/blood-2019-122106

Published: 2019

Journal Articles

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Jean-Sebastien Diana, MD, Naim Bouazza, PhD, Chloé Couzin, MD, Martin Castelle, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Joseph, MD, Marianne Delville, MD, Francois Lefrere, MD, jean-Marc Treluyer, MD PhD, Capucine Picard, MD PhD, Despina Moshous, MD PhD, Isabelle André, PhD, Stephane Blanche, MD, Benedicte Neven, MD PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 5668.

https://doi.org/10.1182/blood-2019-127974

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

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

John E.J. Rasko, MBBS, PhD, Alexis A. Thompson, MD MPH, Janet L. Kwiatkowski, MD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Ying Chen, PhD, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 167.

https://doi.org/10.1182/blood-2018-167

Published: 2018

Journal Articles

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Blood (2017) 130 (Supplement 1): 4609.

https://doi.org/10.1182/blood.V130.Suppl_1.4609.4609

Published: 2017

Journal Articles

Primary Antibody Deficiencies: Scientific Discoveries Leading to Challenging Paradigms in a Complex Disease Landscape

Anne Durandy, MD PhD, Sven Kracker, PhD, Marina Cavazzana, MD PhD

Blood (2017) 130 (Supplement 1): SCI-28.

https://doi.org/10.1182/blood.V130.Suppl_1.SCI-28.SCI-28

Published: 2017

Journal Articles

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Janet L. Kwiatkowski, MD, Alexis A Thompson, MD MPH, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, Marina Cavazzana, MD PhD, Phoebe Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2017) 130 (Supplement 1): 360.

https://doi.org/10.1182/blood.V130.Suppl_1.360.360

Published: 2017

Journal Articles

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Blood (2017) 130 (Supplement 1): 4618.

https://doi.org/10.1182/blood.V130.Suppl_1.4618.4618

Published: 2017

Journal Articles

Gene Therapy Studies in Hemoglobinopathies: Successes and Challenges

Marina Cavazzana, MD PhD

Blood (2016) 128 (22): SCI-50.

https://doi.org/10.1182/blood.V128.22.SCI-50.SCI-50

Published: 2016

Journal Articles

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Alexis A. Thompson, MD MPH, Janet Kwiatkowski, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD FAAP, MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Alexandria Petrusich, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2016) 128 (22): 1175.

https://doi.org/10.1182/blood.V128.22.1175.1175

Published: 2016

Journal Articles

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew M. Hsieh, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Marcelyne Joseney-Antoine, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, John F. Tisdale, MD

Blood (2016) 128 (22): 1176.

https://doi.org/10.1182/blood.V128.22.1176.1176

Published: 2016

Journal Articles

Hemoglobin S Screening Using the « Sickle Scan » - Biomedomics System. the Necker-Enfants Malades Hospital Experience

Louis MINE, Thao Nguyen-Khoa, Birch Allaf, Jean-Antoine Ribeil, MD PhD, Christelle Remus, Valérie Gauthereau, Sarah Enouz, Jason S. KIM, Xiaoxi Yang, Eliane Gluckman, MD PhD, Arnold Munnich, Marina Cavazzana, MD PhD, Robert Girot, MD

Blood (2016) 128 (22): 1308.

https://doi.org/10.1182/blood.V128.22.1308.1308

Published: 2016

Journal Articles

Hydroxycarbamide (HU), Sickle Cell Disease and Pregnancy, a Multicentric Retrospective Study

Laure Joseph, MD, Anoosha Habibi, MD, Giovanna Cannas, MD, Stephanie Ngo, MD, Sylvain Le Jeune, MD, Ersi Voskaridou, MD, Elena Foïs, MD, Caroline Makowski, MD, Nathalie Lemonne, MD, Narcisse Elenga, MD, Yves Ville, MD PhD, Alexandra Benachi, MDPhD, Robert Girot, MD, Marina Cavazzana, MD PhD, Jean-Antoine Ribeil, MD PhD

Blood (2016) 128 (22): 1309.

https://doi.org/10.1182/blood.V128.22.1309.1309

Published: 2016

Journal Articles

Contemporary Conditioning Regimen before Allogeneic Stem Cell Transplantation for Children with Non-Malignant Diseases

Christina Peters, MD, Mary Slatter, MD, Paul Veys, MD Prof., Franco Locatelli, MD, Adriana Balduzzi, MD, M. Akif Yesilipek, Prof. Dr., Jean-Hugues Dalle, MD PhD Prof., Marina Cavazzana, MD PhD, Jaap Jan Boelens, MD PhD, Isaac Yaniv, MD Prof., Ansgar S Schulz, MD PhD Prof., Jacek Wachowiak, Prof MD PhD, Manfred Hoenig, MD, Arjan C Lankester, MD PhD, Benedicte Neven, MD PhD, Eric Beohou, Arnaud Dallisier, Peter Bader, MD, Andrew Gennery, MD

Blood (2016) 128 (22): 3398.

https://doi.org/10.1182/blood.V128.22.3398.3398

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

NARROW

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

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Therapeutic Plasma Exchange in Pediatrics for Immunologic Disorders; Tolerated and Safe Process for Pediatric Life-Threatening Conditions

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

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

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Primary Antibody Deficiencies: Scientific Discoveries Leading to Challenging Paradigms in a Complex Disease Landscape

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Gene Therapy Studies in Hemoglobinopathies: Successes and Challenges

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Hemoglobin S Screening Using the « Sickle Scan » - Biomedomics System. the Necker-Enfants Malades Hospital Experience

Hydroxycarbamide (HU), Sickle Cell Disease and Pregnancy, a Multicentric Retrospective Study

Contemporary Conditioning Regimen before Allogeneic Stem Cell Transplantation for Children with Non-Malignant Diseases

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

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