S. Hacein-Bey-Abina

发表

Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

A. Fischer, W. Kuis, M. Cavazzana‐Calvo, 2002, The New England journal of medicine.

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

F. Bushman, A. Fischer, F. Sigaux, 2008, The Journal of clinical investigation.

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

Manfred Schmidt, Philippe Leboulch, Cynthia C. Bartholomae, 2009, Science.

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Jérôme Larghero, Kenneth Cornetta, Philippe Leboulch, 2010, Nature.

Gene therapy for severe combined immunodeficiencies and beyond

A. Fischer, S. Hacein-Bey-Abina, 2019, The Journal of experimental medicine.

How Primary Immunodeficiencies Have Made Gene Therapy a Reality

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2014 .

20 years of gene therapy for SCID

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2010, Nature Immunology.

Gene therapy for immunodeficiency diseases.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2004, Seminars in hematology.

Correction of Genetic Blood Defects by Gene Transfer

M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2001 .

Correction of genetic blood defects by gene transfer

M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2001, Current opinion in hematology.

Efficacy of gene therapy for X-linked severe combined immunodeficiency.

Frederic D. Bushman, Lily E. Leiva, Capucine Picard, 2010, The New England journal of medicine.

lymphoblastic leukemia B-cell precursor population initiates acute-/-in a Rag 1 p 19 Arf Loss of

A. Fischer, C. Mullighan, A. Borkhardt, 2011 .

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

A. Fischer, C. Mullighan, A. Borkhardt, 2011, Blood.

Gene therapy for severe combined immunodeficiency.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2005, Annual review of medicine.

Stem cell self-renewal: lessons from bone marrow, gut and iPS toward clinical applications

Chad A. Cowan, C. Mummery, S. Karlsson, 2011, Leukemia.

Failure of SCID-X1 gene therapy in older patients.

Kathryn L. Parsley, C. von Kalle, A. Fischer, 2005, Blood.

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

F. Bushman, A. Fischer, M. Cavazzana‐Calvo, 2011, Blood.

A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

Shoshannah L. Roth, F. Bushman, C. Berry, 2011, Nucleic acids research.

Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype.

A. Fischer, R. Cheynier, M. Cavazzana‐Calvo, 2007, Blood.

Gene therapy for primary immunodeficiencies.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2010, Immunology and allergy clinics of North America.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.

C. von Kalle, A. Fischer, Claudia Cattoglio, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Anti-RNP positivity in primary Sjögren’s syndrome is associated with a more active disease and a more frequent muscular and pulmonary involvement

X. Mariette, R. Seror, G. Nocturne, 2019, RMD Open.

Advances in adoptive immunotherapy to accelerate T-cellular immune reconstitution after HLA-incompatible hematopoietic stem cell transplantation.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2010, Immunotherapy.

The Challenge in Hematopoietic Stem Cell Transplantation: Shortening the Immunodeficiency Period

A. Fischer, Y. Hamel, M. Cavazzana‐Calvo, 2010 .

Immune reconstitution after haematopoietic stem cell transplantation: obstacles and anticipated progress.

A. Fischer, M. Cavazzana‐Calvo, B. Neven, 2009, Current opinion in immunology.

Real-Time Definition of Non-Randomness in the Distribution of Genomic Events

C. von Kalle, A. Fischer, M. Cavazzana‐Calvo, 2007, PloS one.

[Gene therapy of severe combined immunodeficiency disease: proof of principle of efficiency and safety issues. Gene therapy, primary immunodeficiencies, retrovirus, lentivirus, genome].

A. Fischer, S. Hacein-Bey-Abina, A. Garrigue, 2005, Bulletin de l'Academie nationale de medecine.

Erythropoietin is a major regulator of thrombopoiesis in thrombopoietin- dependent and -independent context.

H. Echchakir, S. Hacein-Bey-Abina, Magali Pederzoli-Ribeil, 2020, Experimental hematology.

Intestinal barrier dysfunction plays an integral role in arthritis pathology and can be targeted to ameliorate disease

M. Menon, E. Rosser, N. Klein, 2021, Med.

706. Retrovirus integration site distribution in long-term gene-corrected hematopoiesis of the SCID-X1 gene therapy clinical trial

A. Fischer, F. Ledeist, M. Cavazzana‐Calvo, 2004 .

Articular manifestations in patients with inflammatory bowel diseases treated with anti-TNF

X. Mariette, F. Carbonnel, R. Seror, 2022, RMD Open.

[Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector].

C. von Kalle, A. Fischer, M. Cavazzana‐Calvo, 2010, Bulletin de l'Academie nationale de medecine.

Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients.

A. Fischer, M. Forveille, A. Durandy, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors

A. Fischer, F. Deist, J. Casanova, 2004, Bone Marrow Transplantation.

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

F. Bushman, A. Fischer, C. Berry, 2008, Nucleic acids research.

New insights in COVID-19–associated chilblains: A comparative study with chilblain lupus erythematosus

C. Charpentier, A. Couvelard, D. Descamps, 2020, Journal of the American Academy of Dermatology.

Etanercept concentration and immunogenicity do not influence the response to Etanercept in patients with juvenile idiopathic arthritis.

S. Urien, B. Bader-Meunier, A. Belot, 2019, Seminars in arthritis and rheumatism.

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

A. Fischer, C. Picard, P. Frange, 2015, Blood.

Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

A. Schambach, D. Williams, M. Cavazzana‐Calvo, 2012, Gene Therapy.

Gene therapy for primary immunodeficiencies.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2011, Hematology/oncology clinics of North America.

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

A. Fischer, J. Casanova, C. Picard, 2009, Blood.

Gene therapy of metabolic diseases

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2006, Journal of Inherited Metabolic Disease.

[Gene therapy of children with X-linked severe combined immune deficiency: efficiency and complications].

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2004, Medecine sciences : M/S.

Gene therapy of X-linked severe combined immunodeficiency

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2002, Current opinion in allergy and clinical immunology.

Gene Therapy of X-Linked Severe Combined Immunodeficiency

A. Fischer, S. Hacein-Bey-Abina, Marina Cavazzana-Calvoa, 2002, International journal of hematology.

Low Percentage of Signal Regulatory Protein α/β+ Memory B Cells in Blood Predicts Development of Anti-drug Antibodies (ADA) in Adalimumab-Treated Rheumatoid Arthritis Patients

D. Isenberg, X. Mariette, M. Pallardy, 2018, Front. Immunol..

Gene therapy for primary immunodeficiencies: Part 1.

A. Fischer, M. Cavazzana‐Calvo, A. Aiuti, 2012, Current opinion in immunology.

Loss of p19Arf in a Rag1 (cid:1) / (cid:1) B-cell precursor population initiates acute B-lymphoblastic leukemia

A. Borkhardt, N. Brousse, M. Cavazzana‐Calvo, 2011 .

Improving immune reconstitution while preventing graft-versus-host disease in allogeneic stem cell transplantation.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2002, Seminars in hematology.

Combination of thermal ablation by focused ultrasound, pFAR4-IL-12 transfection and lipidic adjuvant provide a distal immune response

D. Scherman, B. Larrat, V. Escriou, 2022, Exploration of targeted anti-tumor therapy.

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

E. Macintyre, F. Adam, A. Kauskot, 2022, Nature medicine.

Assessing the effect of genetic markers on drug immunogenicity from a mechanistic model-based approach

X. Mariette, M. Pallardy, P. Broët, 2020, BMC Medical Research Methodology.

Articular manifestations in patients with inflammatory bowel diseases treated with anti-TNF

X. Mariette, F. Carbonnel, R. Seror, 2022, RMD Open.

Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study

A. Fischer, F. Deist, M. Cavazzana‐Calvo, 2002, The Lancet.

Fragile sites are preferential targets for integrations of MLV vectors in gene therapy

B. Kerem, M. Cavazzana‐Calvo, A. Bester, 2006, Gene Therapy.

Gene therapy for X-linked severe combined immunodeficiency and other hereditary diseases

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2013 .

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy.

Bruce Aronow, Christof von Kalle, Jean Weissenbach, 2007, The Journal of clinical investigation.

LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Cameron S. Osborne, A. Fischer, C. von Kalle, 2003, Science.

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

S. Hacein-Bey-Abina, E. Six, I. André-Schmutz, 2016, Human gene therapy.

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.

Christof von Kalle, Manfred Schmidt, Salima Hacein-Bey-Abina, 2003, The New England journal of medicine.

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

Christopher Baum, David A. Williams, Gregory Hopkins, 2014, The New England journal of medicine.

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency

David A. Williams, Erik L. Clarke, F. Bushman, 2018, Genome Medicine.

Gene Therapy in Patients with Transfusion‐Dependent β‐Thalassemia

C. von Kalle, J. Rasko, D. Teachey, 2018, The New England journal of medicine.

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes

Kyle Bittinger, Anatoly Dryga, Frederic D. Bushman, 2016, Molecular therapy. Methods & clinical development.

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

Kyle Bittinger, Anatoly Dryga, Frederic D. Bushman, 2016, Molecular therapy. Methods & clinical development.

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

Frederic D. Bushman, Philippe Leboulch, Nirav Malani, 2010, Blood.

Advances in CAR-T Cell Genetic Engineering Strategies to Overcome Hurdles in Solid Tumors Treatment

S. Hacein-Bey-Abina, G. Sarrabayrouse, Stéphanie Bessoles, 2022, Frontiers in Immunology.

Engineering Next-Generation CAR-T Cells for Better Toxicity Management

S. Hacein-Bey-Abina, Stéphanie Bessoles, Alain E Andrea, 2020, International journal of molecular sciences.

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

T. Lefèbvre, H. Puy, O. Hermine, 2017 .

Gene Therapy in a Patient with Sickle Cell Disease: Brief Report

G. Veres, T. Lefèbvre, H. Puy, 2017, The New England journal of medicine.

Immune response profiling of patients with spondyloarthritis reveals signalling networks mediating TNF-blocker function in vivo

M. Dougados, V. Guillemot, F. Berenbaum, 2020, Annals of the Rheumatic Diseases.

Site- and allele-specific polycomb dysregulation in T-cell leukaemia

B. Nadel, M. Gut, I. Gut, 2015, Nature Communications.

Activation-induced cytidine deaminase prevents pro-B cell acute lymphoblastic leukemia by functioning as a negative regulator in Rag1 deficient pro-B cells

Stefan Pinkert, A. Borkhardt, I. Sánchez-García, 2017, Oncotarget.

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

A. Schambach, A. Fischer, A. Galy, 2019, Molecular therapy. Methods & clinical development.

Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

A. Schambach, M. Cavazzana‐Calvo, F. Staal, 2011, Leukemia.

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

F. Bushman, A. Fischer, J. Everett, 2020, Blood.

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial.

C. von Kalle, A. Fischer, M. Cavazzana‐Calvo, 2005, Blood.

Severe combined immunodeficiency. A model disease for molecular immunology and therapy

A. Fischer, M. Cavazzana‐Calvo, G. de Saint Basile, 2005, Immunological reviews.

Inflammatory demyelinating polyneuropathies and lymphoma: clues to diagnosis and therapy

O. Lambotte, C. Goujard, D. Adams, 2021, Leukemia & lymphoma.

Treatment of an infant with X-linked severe combined immunodeficiency ( SCID-X 1 ) by gene therapy in Australia

A. Fischer, M. Cavazzana‐Calvo, I. Alexander, 2000 .

Gene therapy for inherited immunodeficiency

A. Fischer, S. Hacein-Bey-Abina, M. Cavazzana, 2014, Expert opinion on biological therapy.

Specific T cells for the treatment of cytomegalovirus and/or adenovirus in the context of hematopoietic stem cell transplantation.

C. Picard, P. Chevallier, C. Elie, 2016, The Journal of allergy and clinical immunology.

Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID).

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2012, Methods in enzymology.

Gene therapy for primary adaptive immune deficiencies.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2011, The Journal of allergy and clinical immunology.

Long‐term outcome of paediatric anti‐N‐methyl‐D‐aspartate receptor encephalitis

N. Tchitchek, H. Maurey, E. Cheuret, 2022, Developmental medicine and child neurology.

Cytokines and culture medium have a major impact on human in vitro T-cell differentiation.

D. Bonhomme, M. Cavazzana‐Calvo, K. Beldjord, 2011, Blood cells, molecules & diseases.

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

L. Kiger, P. Touraine, F. Monpoux, 2022, Nature Medicine.

1 Gene therapy of primary T cell immunodeficiencies

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2017 .

Gene therapy of primary T cell immunodeficiencies.

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2013, Gene.

The rationale for gene therapy in primary T cell immunodeficiencies

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2013 .

Erratum: Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy (New England Journal of Medicine (April 18, 2002) 346 (1185-1193))

Aj Thrasher, Lily E. Leiva, F Le Deist, 2002 .

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.

A. Fischer, M. Cavazzana‐Calvo, P. Bougnères, 2012, Methods in Enzymology.

Long-term follow-up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

C. Adamsbaum, P. Bougnères, Manfred Schmidt, 2021, Human gene therapy.

Anti-Ma2 antibody encephalitis associated with Sjogren's syndrome.

S. Hacein-Bey-Abina, F. Benoudiba, P. Chrétien, 2021, La Revue de medecine interne.

Regulatory T Cells Increase After rh-MOG Stimulation in Non-Relapsing but Decrease in Relapsing MOG Antibody-Associated Disease at Onset in Children

X. Mariette, C. Leroy, C. Serguera, 2021, Frontiers in Immunology.

Methotrexate and BAFF interaction prevents immunization against TNF inhibitors

P. Dönnes, X. Mariette, P. Roques, 2018, Annals of the rheumatic diseases.

Early blood neurofilament light chain and myelin oligodendrocyte glycoprotein antibody levels associate with different disease courses of myelin oligodendrocyte glycoprotein-associated disease in children

C. Leroy, P. Horellou, K. Deiva, 2023, Brain communications.

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

E. Macintyre, A. Kauskot, A. Toubert, 2022, Nature Medicine.

Incidence and risk factors for adalimumab and infliximab anti-drug antibodies in rheumatoid arthritis: A European retrospective multicohort analysis.

P. Dönnes, C. Wijbrandts, P. Tak, 2019, Seminars in arthritis and rheumatism.

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

David A. Williams, Kathryn L. Parsley, F. Bushman, 2011 .

Gene therapy of X-linked severe combined immunodeficiency.

M. Cavazzana‐Calvo, G. de Saint Basile, S. Hacein-Bey-Abina, 2003, Methods in molecular biology.

Treatment of an infant with X‐linked severe combined immunodeficiency (SCID‐X1) by gene therapy in Australia

A. Fischer, M. Cavazzana‐Calvo, I. Alexander, 2005, The Medical journal of Australia.

Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID).

A. Fischer, M. Cavazzana‐Calvo, S. Hacein-Bey-Abina, 2012, Methods in enzymology.

Flare of a mixed cryoglobulinaemic vasculitis after obinutuzumab infusion.

X. Mariette, G. Nocturne, S. Hacein-Bey-Abina, 2021, Clinical and experimental rheumatology.

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

A. Fischer, C. Mullighan, A. Borkhardt, 2011, Blood.

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.

A. Fischer, M. Cavazzana‐Calvo, P. Bougnères, 2012, Methods in enzymology.

Erythropoietin induces tumour progression and CD39 expression on immune cells in a preclinical model of triple-negative breast cancer.

P. de la Grange, S. Hacein-Bey-Abina, Amine M Abina, 2024, Immunology.

Discovery of Anti-SS-A Antibodies during Stroke Investigations in Young Adults: What Impact?

S. Hacein-Bey-Abina, N. Legris, S. Henriquez, 2021, Journal of stroke and cerebrovascular diseases : the official journal of National Stroke Association.