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Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Blood (2021) 138 (Supplement 1): 2934.

https://doi.org/10.1182/blood-2021-147697

Published: 2021

Journal Articles

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Blood (2020) 135 (15): 1219–1231.

https://doi.org/10.1182/blood.2019002350

Published: 2020

Includes: Supplemental data

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

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Blood (2016) 128 (22): 2311.

https://doi.org/10.1182/blood.V128.22.2311.2311

Published: 2016

Journal Articles

a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients

Emmanuelle Six, PhD, Laure Caccavelli, Arnaud Lecoules, Christopher L Nobles, PhD, France Male, Cécile Roudaut, MSc, Clemence Plantier, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Nicolas Cagnard, Anne Galy, PharmD, PhD, Adrian J Thrasher, MD PhD, Salima hacein-Bey Abina, Agathe Guilloux, Isabelle Andre-Schmutz, Charles C. Berry, frederic D. Bushman, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 3871.

https://doi.org/10.1182/blood.V128.22.3871.3871

Published: 2016

Journal Articles

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

Fabien Touzot, Despina Moshous, Rita Creidy, Bénédicte Neven, Pierre Frange, Guilhem Cros, Laure Caccavelli, Johanna Blondeau, Alessandra Magnani, Jean-Marc Luby, Brigitte Ternaux, Capucine Picard, Stéphane Blanche, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana

Blood (2015) 125 (23): 3563–3569.

https://doi.org/10.1182/blood-2014-12-616003

Published: 2015

Includes: Supplemental data

Journal Articles

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Blood (2014) 124 (21): 4797.

https://doi.org/10.1182/blood.V124.21.4797.4797

Published: 2014

Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Blood (2013) 122 (21): 715.

https://doi.org/10.1182/blood.V122.21.715.715

Published: 2013

Journal Articles

Anti CMV and/or Anti Adenovirus IFN-g-Positive CD4+ CD8+ T Lymphocytes for Treatment of Viral Infections After Allogeneic HSC Transplantation: First Results

Liliane Dal Cortivo, MD, RITA Creidy, PhD, Aurélie Gabrion, Marianne Leruez-Ville, Sebastien Heritier, MD, Fabien Touzot, MD, PhD, Guilhem Cros, MD, Sebastien Maury, MD, Alienor Xhaard, Marie Ouachee, MD, Ingrid Hamon, Tech, Capucine Picard, MD, PhD, Christophe Cambouris, Salima Hacein Bey Abina, PhD, Stephane Blanche, MD, Marina Cavazzana-Calvo, MD, PhD

Blood (2012) 120 (21): 1906.

https://doi.org/10.1182/blood.V120.21.1906.1906

Published: 2012

Journal Articles

Massive expansion of maternal T cells in response to EBV infection in a patient with SCID-Xl

Fabien Touzot, Liliane Dal-Cortivo, Virginie Verkarre, Annick Lim, Anne Crucis-Armengaud, Despina Moshous, Sébastien Héritier, Pierre Frange, Sophie Kaltenbach, Stéphane Blanche, Capucine Picard, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Alain Fischer

Blood (2012) 120 (9): 1957–1959.

https://doi.org/10.1182/blood-2012-04-426833

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

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Blood (2011) 118 (21): 164.

https://doi.org/10.1182/blood.V118.21.164.164

Published: 2011

Journal Articles

Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in X-Linked Adrenoleukodystrophy

Patrick Aubourg, MD, Salima Hacein-Bey-Abina, PharmD, PhD, Cynthia Bartholomae, PhD, Manfred Schmidt, PhD, Ina Kutschera, PhD, Michel Vidaud, PharmD, PhD, Liliane Dal-Cortivo, MD, PhD, Nizar Mahlaoui, MD, Pierre Frange, MD, Céline Bellesme, MD, Francois Lefrère, MD, Gaelle Dufayet, Alain Fischer, MD/PhD, Christof van Kalle, MD, PhD, Marina Cavazzana-Calvo, MD, PhD, Nathalie Cartier, MD, PhD

Blood (2011) 118 (21): 163.

https://doi.org/10.1182/blood.V118.21.163.163

Published: 2011

Journal Articles

Loss of p19Arf in a Rag1 −/− B-cell precursor population initiates acute B-lymphoblastic leukemia

Julia Hauer, Charles Mullighan, Estelle Morillon, Gary Wang, Julie Bruneau, Nicole Brousse, Marc Lelorc'h, Serge Romana, Amine Boudil, Daniela Tiedau, Sven Kracker, Frederic D. Bushmann, Arndt Borkhardt, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo

Blood (2011) 118 (3): 544–553.

https://doi.org/10.1182/blood-2010-09-305383

Published: 2011

Includes: Supplemental data

Journal Articles

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

Marina Cavazzana-Calvo, Alain Fischer, Frederic D. Bushman, Emmanuel Payen, Salima Hacein-Bey-Abina, Philippe Leboulch

Blood (2011) 117 (17): 4420–4424.

https://doi.org/10.1182/blood-2010-09-255679

Published: 2011

Journal Articles

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Sheng Zhou, Disha Mody, Suk See DeRavin, Julia Hauer, Taihe Lu, Zhijun Ma, Salima Hacein-Bey Abina, John T. Gray, Michael R. Greene, Marina Cavazzana-Calvo, Harry L. Malech, Brian P. Sorrentino

Blood (2010) 116 (6): 900–908.

https://doi.org/10.1182/blood-2009-10-250209

Published: 2010

Includes: Supplemental data

Journal Articles

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Gary P. Wang, Charles C. Berry, Nirav Malani, Philippe Leboulch, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Frederic D. Bushman

Blood (2010) 115 (22): 4356–4366.

https://doi.org/10.1182/blood-2009-12-257352

Published: 2010

Includes: Supplemental data

Journal Articles

Leukemia Prone B-Precursor Population in a p19ARF-/-RAG1-/- Mouse Model.

Julia Hauer, MD, Charles G. Mullighan, MBBS(Hons), MSc, MD, Estelle Morillon, Gary P Wang, MD, PhD, Julie Bruneau, Nicole Brousse, Arndt Borkhardt, MD, Frederic D Bushmann, PhD, Alain Fischer, MD, PhD, Salima Hacein-Bey-Abina, Pharm.D., Ph.D., Marina Cavazzana-Calvo, MD, PhD

Blood (2009) 114 (22): 3971.

https://doi.org/10.1182/blood.V114.22.3971.3971

Published: 2009

Journal Articles

Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency

Bénédicte Neven, Sandrine Leroy, Hélène Decaluwe, Francoise Le Deist, Capucine Picard, Despina Moshous, Nizar Mahlaoui, Marianne Debré, Jean-Laurent Casanova, Liliane Dal Cortivo, Yoann Madec, Salima Hacein-Bey-Abina, Geneviève de Saint Basile, Jean-Pierre de Villartay, Stéphane Blanche, Marina Cavazzana-Calvo, Alain Fischer

Blood (2009) 113 (17): 4114–4124.

https://doi.org/10.1182/blood-2008-09-177923

Published: 2009

Includes: Supplemental data

Journal Articles

Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector in X-Linked Adrenoleukodystrophy

Blood (2008) 112 (11): 821.

https://doi.org/10.1182/blood.V112.11.821.821

Published: 2008

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Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

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

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Anti CMV and/or Anti Adenovirus IFN-g-Positive CD4+ CD8+ T Lymphocytes for Treatment of Viral Infections After Allogeneic HSC Transplantation: First Results

Massive expansion of maternal T cells in response to EBV infection in a patient with SCID-Xl

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

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in X-Linked Adrenoleukodystrophy

Loss of p19Arf in a Rag1 −/− B-cell precursor population initiates acute B-lymphoblastic leukemia

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Leukemia Prone B-Precursor Population in a p19ARF-/-RAG1-/- Mouse Model.

Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency

Hematopoietic Stem Cell Gene Therapy Trial with Lentiviral Vector in X-Linked Adrenoleukodystrophy

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