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Journal Articles

SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: SI101-02 First-in-Human Phase I/II

Raynier Devillier, Regis Peffault De Latour, Patrice Chevallier, Anne Huynh, Patrizia Chiusolo, Aurelie Bauquet, Laura Simons, Pierre Heimendinger, Olivier Negre, Shabi Soheili, Juliette Paillet, Isabelle Andre, Sebastien Oster, Marina Cavazzana, Frédéric François Lehmann, Fabio Ciceri

Blood (2023) 142 (Supplement 1): 4861.

https://doi.org/10.1182/blood-2023-179215

Published: 2023

Journal Articles

Human Pro-T-Cell Manufacturing in Vitro Is a Safe Procedure for Hematopoietic Stem Cell Transplantation with Delayed T-Cell Reconstitution: Interim Results from 2 Different Clinical Trials

Blood (2023) 142 (Supplement 1): 6868.

https://doi.org/10.1182/blood-2023-187607

Published: 2023

Journal Articles

Multiplex CRISPR/Cas9 Genome Editing Targeting the BCL11A/HBG Axis Maximizes Fetal Hemoglobin Reinduction but Generates Chromosomal Translocations Which Persist In Vivo

Clare Samuelson, Olivier Humbert, PhD, Stefan Radtke, PhD, Haiying Zhu, Mallory J Llewellyn, BS, Emily Fields, BS, Savannah Cook, BS, Meei-Li W Huang, Olivier Negre, PhD, Keith Jerome, MDPhD, Hans-Peter Kiem, MDPhD

Blood (2020) 136 (Supplement 1): 47–48.

https://doi.org/10.1182/blood-2020-138539

Published: 2020

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Blood (2018) 132 (Supplement 1): 1023.

https://doi.org/10.1182/blood-2018-99-116733

Published: 2018

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

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Olivier Humbert, PhD, Stefan Radtke, PhD, Ray R Carillo, Anai M Perez, Sowmya Somashekar Reddy, Lauren E Schefter, Christopher Lux, MDPhD, Sowmya Pattabhi, PhD, Olivier Negre, PhD, Ciaran M Lee, PhD, Jennifer Adair, PhD, Christopher Peterson, PhD, Gang Bao, PhD, David J. Rawlings, MD, Andrew M. Scharenberg, MD, Hans-Peter Kiem, MD PhD

Blood (2018) 132 (Supplement 1): 806.

https://doi.org/10.1182/blood-2018-99-112996

Published: 2018

Journal Articles

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Olivier Negre, PhD, Christian Brendel, PhD, Geoffrey B. Parsons, Min Luo, PhD, Myriam A Armant, PhD, Swaroopa Guda, PhD, Lauryn Christiansen, Gretchen Lewis, BMBS, Gabor Veres, PhD, Melissa Bonner, PhD, David A. Williams, MD

Blood (2017) 130 (Supplement 1): 3557.

https://doi.org/10.1182/blood.V130.Suppl_1.3557.3557

Published: 2017

Journal Articles

Heat Shock Protein 70 Cytosolic Sequestration by Excess of Free Alpha-Globin Chains Is a Key Mechanism of the Ineffective Erythropoiesis in β-Thalassemia Major Patients

Jean-Benoît Arlet, MD, Jean-Antoine Ribeil, MD, PhD, Adonis Hazoumé, PhD, Samuel Demarest, Luba Tchertanov, PhD, Dussiot Michael, Ivan Cruz Moura, PhD, Thiago Maciel, PhD, Christian Auclair, PhD, Olivier Negre, PhD, Philippe Leboulch, MD, Véronique Baudin-creuza, PhD, Carmen Garrido, PhD, Genevieve Courtois, PhD, Olivier Hermine, MD, PhD

Blood (2012) 120 (21): 823.

https://doi.org/10.1182/blood.V120.21.823.823

Published: 2012

Journal Articles

HSC Transplantation for Hemoglobinopathies: Allogeneic or Autologous Gene-Modified HSC?

Marina Cavazzana-Calvo, MD, PhD, Emmanuel Payen, Olivier Negre, PhD, Salima Hacein-Bey-Abina, PharmD, PhD, Philippe Leboulch, PhD

Blood (2011) 118 (21): SCI-46.

https://doi.org/10.1182/blood.V118.21.SCI-46.SCI-46

Published: 2011

Journal Articles

Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

Olivier Negre, Floriane Fusil, Charlotte Colomb, Shoshannah Roth, Beatrix Gillet-Legrand, Annie Henri, Yves Beuzard, Frederic Bushman, Philippe Leboulch, Emmanuel Payen

Blood (2011) 117 (20): 5321–5331.

https://doi.org/10.1182/blood-2010-01-263582

Published: 2011

Includes: Supplemental data

NARROW

SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: SI101-02 First-in-Human Phase I/II

Human Pro-T-Cell Manufacturing in Vitro Is a Safe Procedure for Hematopoietic Stem Cell Transplantation with Delayed T-Cell Reconstitution: Interim Results from 2 Different Clinical Trials

Multiplex CRISPR/Cas9 Genome Editing Targeting the BCL11A/HBG Axis Maximizes Fetal Hemoglobin Reinduction but Generates Chromosomal Translocations Which Persist In Vivo

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

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

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Heat Shock Protein 70 Cytosolic Sequestration by Excess of Free Alpha-Globin Chains Is a Key Mechanism of the Ineffective Erythropoiesis in β-Thalassemia Major Patients

HSC Transplantation for Hemoglobinopathies: Allogeneic or Autologous Gene-Modified HSC?

Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

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American Society of Hematology

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American Society of Hematology

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