Marina Cavazzana | Academic Influence

Marina Cavazzana's AcademicInfluence.com Rankings

Marina Cavazzana

Philosophy

#5662

World Rank

#8529

Historical Rank

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#4875

World Rank

#6207

Historical Rank

philosophy Degrees

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Philosophy

Marina Cavazzana's Degrees

Doctorate Medicine University of Milan
PhD Cellular Biology University of Milan

Why Is Marina Cavazzana Influential?

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According to Wikipedia, Marina Cavazzana is a professor of Paediatric Immunology at the Necker-Enfants Malades Hospital and the Imagine Institute, as well as an academic at Paris Descartes University. She was awarded the Irène Joliot-Curie Prize in 2012 and elected to the National Academy of Medicine in 2019.

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Marina Cavazzana's Published Works

Number of citations in a given year to any of this author's works

Total number of citations to an author for the works they published in a given year. This highlights publication of the most important work(s) by the author

Published Works

LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1 (2003) (3518)
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. (2000) (2630)
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. (2003) (1868)
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. (2008) (1642)
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy (2009) (1367)
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia (2010) (1222)
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. (2002) (1137)
Artemis, a Novel DNA Double-Strand Break Repair/V(D)J Recombination Protein, Is Mutated in Human Severe Combined Immune Deficiency (2001) (886)
Efficacy of gene therapy for X-linked severe combined immunodeficiency. (2010) (581)
Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99 (2003) (532)
Gene Therapy in Patients with Transfusion‐Dependent β‐Thalassemia (2018) (425)
Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? (2010) (386)
International AIDS Society global scientific strategy: towards an HIV cure 2016 (2016) (372)
Gene Therapy in a Patient with Sickle Cell Disease: Brief Report (2017) (346)
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. (2015) (326)
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. (2014) (312)
Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients. (2003) (310)
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. (2017) (307)
Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel (2014) (288)
Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: definitions and current practice in Europe (2002) (275)
The future of gene therapy (2004) (270)
Bone marrow failure in Fanconi anemia is triggered by an exacerbated p53/p21 DNA damage response that impairs hematopoietic stem and progenitor cells. (2012) (265)
X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. (2011) (263)
Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. (1998) (260)
Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. (2011) (254)
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. (2007) (253)
Treatment of the immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) by allogeneic bone marrow transplantation. (2001) (248)
Hematopoietic Stem Cell Transplantation in Hemophagocytic Lymphohistiocytosis: A Single-Center Report of 48 Patients (2006) (239)
Severe combined immunodeficiency. A model disease for molecular immunology and therapy (2005) (236)
New ISSCR guidelines underscore major principles for responsible translational stem cell research. (2008) (230)
Gene therapy for severe combined immunodeficiency: are we there yet? (2007) (225)
Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study (2002) (223)
Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. (2008) (221)
Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency. (2009) (219)
Partial T and B lymphocyte immunodeficiency and predisposition to lymphoma in patients with hypomorphic mutations in Artemis. (2003) (208)
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study (2017) (205)
Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure (2007) (200)
Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness (2009) (196)
Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV (2006) (184)
Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. (2013) (179)
Naturally occurring primary deficiencies of the immune system. (1997) (169)
Clinical and immunologic phenotype associated with activated phosphoinositide 3-kinase δ syndrome 2: A cohort study. (2016) (166)
Gene therapy for severe combined immunodeficiency. (2005) (160)
20 years of gene therapy for SCID (2010) (159)
Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. (2012) (156)
Gene therapy: X-SCID transgene leukaemogenicity (2006) (156)
Severe cutaneous papillomavirus disease after haemopoietic stem-cell transplantation in patients with severe combined immune deficiency caused by common γc cytokine receptor subunit or JAK-3 deficiency (2004) (151)
Human and murine amniotic fluid c-Kit+Lin- cells display hematopoietic activity. (2009) (149)
A Human Severe Combined Immunodeficiency (SCID) Condition with Increased Sensitivity to Ionizing Radiations and Impaired V(D)J Rearrangements Defines a New DNA Recombination/Repair Deficiency (1998) (149)
Severe combined immunodeficiencies and related disorders (2015) (146)
A human immunodeficiency caused by mutations in the PIK3R1 gene. (2014) (136)
Failure of SCID-X1 gene therapy in older patients. (2005) (131)
Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial. (2010) (130)
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene (2016) (127)
Long-term chimerism and B-cell function after bone marrow transplantation in patients with severe combined immunodeficiency with B cells: A single-center study of 22 patients. (1999) (127)
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. (2018) (127)
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. (2007) (126)
Depletion of alloreactive T cells by a specific anti-interleukin-2 receptor p55 chain immunotoxin does not impair in vitro antileukemia and antiviral activity. (1999) (125)
SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. (2014) (124)
Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002. (2003) (122)
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)) (2002) (121)
Severe combined immunodeficiency caused by deficiency in either the δ or the ε subunit of CD3 (2004) (121)
Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency. (1999) (119)
Bone marrow transplantation (BMT) in Europe for primary immunodeficiencies other than severe combined immunodeficiency: a report from the European Group for BMT and the European Group for Immunodeficiency (1994) (118)
Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy. (2012) (117)
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry (2018) (117)
Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. (2009) (117)
Role of interleukin-2 (IL-2), IL-7, and IL-15 in natural killer cell differentiation from cord blood hematopoietic progenitor cells and from gamma c transduced severe combined immunodeficiency X1 bone marrow cells. (1996) (117)
A human postnatal lymphoid progenitor capable of circulating and seeding the thymus (2007) (115)
A human immunodeficiency caused by mutations in the PIK3R1 gene. (2015) (115)
Dynamics of thymus-colonizing cells during human development. (2006) (115)
A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. (2010) (115)
A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome. (2007) (112)
Intensive and Prolonged Treatment of Focal and Segmental Glomerulosclerosis Recurrence in Adult Kidney Transplant Recipients: A Pilot Study (2009) (111)
Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges (2019) (110)
Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? (2012) (109)
Bone marrow-derived mononuclear cell therapy induces distal angiogenesis after local injection in critical leg ischemia (2008) (108)
Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center. (1996) (107)
DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer (2008) (106)
Early posttransplantation donor-derived invariant natural killer T-cell recovery predicts the occurrence of acute graft-versus-host disease and overall survival. (2012) (101)
Results of allogeneic bone marrow transplantation in patients with leukocyte adhesion deficiency. (1995) (101)
gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function. (1996) (100)
Allogeneic bone marrow transplantation in mevalonic aciduria. (2007) (98)
Gene Therapy for β-Hemoglobinopathies. (2017) (96)
Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease (2014) (96)
Gene therapy for primary immunodeficiencies: Part 1. (2012) (95)
Gene therapy of inherited diseases (2008) (95)
Increased radiosensitivity of granulocyte macrophage colony-forming units and skin fibroblasts in human autosomal recessive severe combined immunodeficiency. (1993) (94)
Bone marrow transplantation in major histocompatibility complex class II deficiency: a single-center study of 19 patients. (1995) (92)
Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006). (2007) (91)
Treatment of familial hemophagocytic lymphohistiocytosis with bone marrow transplantation from HLA genetically nonidentical donors. (1997) (88)
Attenuation of graft-versus-host disease and graft rejection by ex vivo immunotoxin elimination of alloreactive T cells in an H-2 haplotype disparate mouse combination. (1994) (85)
ISSCR Guidelines for the Clinical Translation of Stem Cells. (2009) (81)
Gene therapy for primary immunodeficiencies. (2010) (81)
Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome. (2013) (79)
Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. (2002) (79)
Severe combined immunodeficiency caused by deficiency in either the delta or the epsilon subunit of CD3. (2004) (77)
Allogeneic Hematopoietic Stem-Cell Transplantation for Leukocyte Adhesion Deficiency (2009) (76)
Gene therapy of X-linked severe combined immunodeficiency (2002) (76)
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion (2018) (75)
Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial. (2005) (74)
Gene therapy for primary adaptive immune deficiencies. (2011) (74)
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand? (2016) (73)
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer (2011) (70)
A method to sequence and quantify DNA integration for monitoring outcome in gene therapy (2011) (67)
Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease (2016) (67)
Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity. (2006) (66)
Human T-Lymphoid Progenitors Generated in a Feeder-Cell-Free Delta-Like-4 Culture System Promote T-Cell Reconstitution in NOD/SCID/γc−/− Mice (2012) (65)
INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes (2016) (64)
Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies. (2019) (63)
Amniotic Fluid Stem Cells Restore the Muscle Cell Niche in a HSA‐Cre, SmnF7/F7 Mouse Model (2012) (62)
First experience of autologous peripheral blood stem cell mobilization with biosimilar granulocyte colony- stimulating factor (2011) (61)
Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1. (2015) (61)
An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, and MPN domains 1 (MYSM1) in human hematopoiesis and lymphocyte differentiation. (2015) (60)
Characteristics of antigen‐independent and antigen‐dependent interaction of dendritic cells with CD4+ T cells (1995) (60)
Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients. (2009) (60)
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions. (2016) (59)
Gene therapy of primary T cell immunodeficiencies. (2013) (59)
Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice. (2003) (58)
Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells. (2008) (58)
Correction of Fas (CD95) deficiency by haploidentical bone marrow transplantation (1997) (57)
CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: A preliminary study. (2015) (56)
AK2 deficiency compromises the mitochondrial energy metabolism required for differentiation of human neutrophil and lymphoid lineages (2015) (55)
Treatment of Omenn syndrome by bone marrow transplantation. (1995) (54)
Gene therapy for primary immunodeficiencies: Part 2. (2012) (54)
X-linked primary immunodeficiency associated with hemizygous mutations in the moesin (MSN) gene. (2016) (53)
γc Gene Transfer in the Presence of Stem Cell Factor, FLT-3L, Interleukin-7 (IL-7), IL-1, and IL-15 Cytokines Restores T-Cell Differentiation From γc(−) X-Linked Severe Combined Immunodeficiency Hematopoietic Progenitor Cells in Murine Fetal Thymic Organ Cultures (1998) (52)
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome. (2014) (51)
INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions (2016) (50)
Clinical and immunologic outcome of patients with cartilage hair hypoplasia after hematopoietic stem cell transplantation. (2010) (49)
Gene therapy of severe combined immunodeficiencies (2002) (49)
Haematopoietic stem cell transplantation for SCID patients: where do we stand? (2013) (48)
Adenovirally transduced dendritic cells induce bispecific cytotoxic T lymphocyte responses against adenovirus and cytomegalovirus pp65 or against adenovirus and Epstein-Barr virus EBNA3C protein: a novel approach for immunotherapy. (2002) (48)
Gene therapy for inherited immunodeficiency (2014) (48)
Primary Microcephaly, Impaired DNA Replication, and Genomic Instability Caused by Compound Heterozygous ATR Mutations (2013) (48)
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 (2004) (48)
Gene Therapy Approaches to Hemoglobinopathies. (2017) (48)
Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression. (2010) (48)
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype (2020) (47)
Bone marrow transplantation from genetically HLA-nonidentical donors in children with fatal inherited disorders excluding severe combined immunodeficiencies: use of two monoclonal antibodies to prevent graft rejection. (1996) (47)
Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives (2019) (46)
Treatment of an infant with X‐linked severe combined immunodeficiency (SCID‐X1) by gene therapy in Australia (2005) (46)
Gene therapy for human severe combined immunodeficiencies. (2001) (46)
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. (2011) (45)
LMO2 and gene therapy for severe combined immunodeficiency. (2004) (45)
Fragile sites are preferential targets for integrations of MLV vectors in gene therapy (2006) (44)
Bone marrow transplantation for primary immunodeficiency diseases. (2010) (44)
Clinical and immunologic phenotype associated with activated ă phosphoinositide 3-kinase delta syndrome 2: A cohort study (2016) (44)
Bone marrow transplantation (BMT) in Europe for primary immunodeficiencies other than severe combined immunodeficiency: a report from the European Group for BMT and the European Group for Immunodeficiency. (1994) (44)
Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome (2017) (42)
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. (2011) (42)
Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients. (2008) (41)
Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease (2019) (41)
A phase II trial of partially incompatible bone marrow transplantation for high‐risk acute lymphoblastic leukaemia in children: prevention of graft rejection with anti‐LFA‐1 and anti‐CD2 antibodies (1996) (40)
Human leucocyte antigen‐identical haematopoietic stem cell transplantation in major histocompatiblity complex class II immunodeficiency: reduced survival correlates with an increased incidence of acute graft‐versus‐host disease and pre‐existing viral infections (2006) (40)
Is normal hematopoiesis maintained solely by long-term multipotent stem cells? (2011) (40)
Induction of NKG2D ligands by gamma radiation and tumor necrosis factor-alpha may participate in the tissue damage during acute graft-versus-host disease. (2008) (39)
Gene therapy of hemoglobinopathies: progress and future challenges. (2019) (39)
Immune reconstitution after haematopoietic stem cell transplantation: obstacles and anticipated progress. (2009) (39)
Efficacy of gene therapy for SCID is being confirmed (2004) (38)
Treatment of Familial Hemophagocytic Lymphohistiocytosis With Bone Marrow Transplantation From HLA Genetically Nonidentical Donors (1997) (38)
Lymphoid-affiliated genes are associated with active histone modifications in human hematopoietic stem cells. (2008) (38)
Treatment of Familial Hemophagocytic Lymphohistiocytosis With Bone Marrow Transplantation From HLA Genetically Nonidentical Donors (1997) (38)
Real-Time Definition of Non-Randomness in the Distribution of Genomic Events (2007) (37)
Pre-transplant donor CD4− invariant NKT cell expansion capacity predicts the occurrence of acute graft-versus-host disease (2017) (37)
Failure of bone marrow transplantation to eradicate HIV reservoir despite efficient HAART. (2007) (36)
An inherited immunoglobulin class-switch recombination deficiency associated with a defect in the INO80 chromatin remodeling complex (2015) (36)
Evaluation of an algorithm based on peripheral blood hematopoietic progenitor cell and CD34+ cell concentrations to optimize peripheral blood progenitor cell collection by apheresis (2007) (34)
Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs. (2020) (34)
Mesenchymal stromal cells can be derived from bone marrow CD133+ cells: implications for therapy. (2009) (34)
A specific time course for mobilization of peripheral blood CD34+ cells after plerixafor injection in very poor mobilizer patients: impact on the timing of the apheresis procedure (2013) (33)
Gene Therapy in a Patient with Sickle Cell Disease. (2017) (33)
Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia: Analysis of cost-effectiveness based on French real field data. (2015) (33)
Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product) (2014) (33)
Comparison of lenograstim vs filgrastim administration following chemotherapy for peripheral blood stem cell (PBSC) collection: a retrospective study of 126 patients. (1999) (32)
Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector (2015) (32)
Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells. (2001) (31)
Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination. (2013) (31)
Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation. (2019) (31)
Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression. (2010) (30)
Integration of Retroviruses: A Fine Balance between Efficiency and Danger (2005) (30)
Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome. (2017) (30)
Cytokines and culture medium have a major impact on human in vitro T-cell differentiation. (2011) (30)
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome (2022) (29)
Loss of ARHGEF1 causes a human primary antibody deficiency (2019) (28)
Improving immune reconstitution while preventing graft-versus-host disease in allogeneic stem cell transplantation. (2002) (27)
Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study. (2019) (27)
The BLNK adaptor protein has a nonredundant role in human B-cell differentiation. (2014) (27)
Mammalian target of rapamycin inhibition counterbalances the inflammatory status of immune cells in patients with chronic granulomatous disease (2017) (26)
[Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector]. (2010) (26)
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency (2018) (26)
Mutations in the adaptor-binding domain and associated linker region of p110δ cause Activated PI3K-δ Syndrome 1 (APDS1) (2017) (26)
Bone Marrow Transplantation Attenuates the Myopathic Phenotype of a Muscular Mouse Model of Spinal Muscular Atrophy (2006) (25)
Gene therapy of severe combined immunodeficiencies (2001) (25)
The VAD chemotherapy regimen plus a G-CSF dose of 10 μg/kg is as effective and less toxic than high-dose cyclophosphamide plus a G-CSF dose of 5 μg/kg for progenitor cell mobilization: results from a monocentric study of 82 patients (2006) (25)
A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells (2018) (25)
Gene Therapy for Hemoglobinopathies (2018) (25)
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID (2020) (24)
Treatment of juvenile chronic myelomonocytic leukemia by allogeneic bone marrow transplantation. (1994) (24)
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial (2022) (24)
Blockade of the integrin αLβ2 but not of integrins α4 and/or β7 significantly prolongs intestinal allograft survival in mice (2000) (24)
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype (2020) (23)
Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study (2016) (22)
Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy (2019) (22)
Improving immune reconstitution while preventing GvHD in allogeneic stem cell transplantation. (2005) (22)
CD34+ stem cell top‐ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies (2006) (22)
Gene Therapy of the b-Hemoglobinopathies by Lentiviral Transfer of the b A ( T 87 Q )-Globin Gene (2016) (22)
Specific T cells for the treatment of cytomegalovirus and/or adenovirus in the context of hematopoietic stem cell transplantation. (2016) (22)
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View. (2017) (22)
Functional study of residual T lymphocytes after specific elimination of alloreactive T cells by a specific anti-interleukin-2 receptor Bk chain immunotoxin. (1993) (21)
Successful haematopoietic stem cell transplantation in a case of pulmonary alveolar proteinosis due to GM-CSF receptor deficiency (2017) (21)
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency (2016) (21)
Donor T lymphocyte infusion following ex vivo depletion of donor anti-host reactivity by a specific anti-interleukin-2 receptor P55 chain immunotoxin. (2002) (21)
Gene therapy of severe combined immunodeficiencies (2000) (21)
Successful peripheral blood stem cell harvesting with granulocyte colony-stimulating factor alone after previous mobilization failure. (2004) (20)
Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced dysfunction (2021) (20)
Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice. (2016) (19)
Gene therapy for primary immunodeficiencies (2015) (19)
Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency. (2019) (18)
Competition within the early B-cell compartment conditions B-cell reconstitution after hematopoietic stem cell transplantation in nonirradiated recipients. (2006) (18)
IL‐7 effect on immunological reconstitution after HSCT depends on MHC incompatibility (2004) (18)
Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach (2016) (18)
Hematopoietic cell transplantation in severe combined immunodeficiency: the SCETIDE 2006-2014 European cohort. (2021) (18)
Massive expansion of maternal T cells in response to EBV infection in a patient with SCID-Xl. (2012) (17)
Generation of adult human T-cell progenitors for immunotherapeutic applications (2017) (17)
Retrieval of vector integration sites from cell-free DNA (2021) (17)
Successful RAG1-SCID gene therapy depends on the level of RAG1 expression. (2014) (17)
Trisomy 21 mid-trimester amniotic fluid induced pluripotent stem cells maintain genetic signatures during reprogramming: implications for disease modeling and cryobanking. (2014) (17)
Single-cell analysis of FOXP3 deficiencies in humans and mice unmasks intrinsic and extrinsic CD4+ T cell perturbations (2020) (17)
Characterization of antigen‐specific repertoire diversity following in vitro restimulation by a recombinant adenovirus expressing human cytomegalovirus pp65 (2003) (16)
[Gene therapy of severe combined immunodeficiencies]. (2000) (16)
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype (2018) (16)
Arterio‐venous fistula for automated red blood cells exchange in patients with sickle cell disease: Complications and outcomes (2017) (16)
Occurrence of myelodysplastic syndrome in 2 patients with reticular dysgenesis. (2011) (15)
Gene therapy of X-linked severe combined immunodeficiency. (2003) (15)
Comparing genotoxic signatures in cord blood cells from neonates exposed in utero to zidovudine or tenofovir (2015) (15)
Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells (2014) (15)
Gene therapy for immunodeficiency diseases. (2004) (15)
Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease (2015) (14)
Loss of p19Arf in a Rag1(-/-) B-cell precursor population initiates acute B-lymphoblastic leukemia. (2011) (14)
Gene Therapy for b-Hemoglobinopathies (14)
Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency. (2017) (14)
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID (2019) (14)
gammac gene transfer in the presence of stem cell factor, FLT-3L, interleukin-7 (IL-7), IL-1, and IL-15 cytokines restores T-cell differentiation from gammac(-) X-linked severe combined immunodeficiency hematopoietic progenitor cells in murine fetal thymic organ cultures. (1998) (14)
Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βΑ-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product) (2014) (14)
Correction of genetic blood defects by gene transfer (2001) (13)
Concise Review: Boosting T‐Cell Reconstitution Following Allogeneic Transplantation—Current Concepts and Future Perspectives (2019) (13)
Stem cell transplantation for primary immunodeficiencies: the European experience (2014) (13)
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Two Monogenetic Disorders, Activated PI3-Kinase-δ Syndrome 2 and Smith–Magenis Syndrome, in One Patient: Case Report and a Literature Review of Neurodevelopmental Impact in Primary Immunodeficiencies Associated With Disturbed PI3K Signaling (2021) (1)
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T-Cell Progenitors As A New Immunotherapy to Bypass Hurdles of Allogeneic Hematopoietic Stem Cell Transplantation (2022) (1)
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Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up (2021) (1)
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Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes (2019) (0)
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Progrès de la thérapie génique dans les maladies génétiques du système hématopoïétique (2022) (0)
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Efficacité et sécurité du traitement par thérapie génique des patients atteints du syndrome de Wiskott-Aldrich (2015) (0)
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Contemporary Conditioning Regimen before Allogeneic Stem Cell Transplantation for Children with Non-Malignant Diseases (2016) (0)
The Challenge in Hematopoietic Stem Cell Transplantation: Shortening the Immunodeficiency Period (2010) (0)
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency (2018) (0)
CD34+stem cell top-ups without conditioning after initial HSCT for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies (2006) (0)
S141 CLINICAL OUTCOMES OF LENTIGLOBIN GENE THERAPY FOR TRANSFUSION-DEPENDENT β-THALASSAEMIA (TDT) FOLLOWING COMPLETION OF THE NORTHSTAR (HGB-204) STUDY (2019) (0)
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Radiation-Free Salvage Haploidentical HSCT with Post Tx Cy after Rejection/Graft Failure in Children with Non-Malignant Disorders (2017) (0)
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therapy trial differentiation potential and self-renewal capacity in the SCID-X1 gene Clonal evidence for the transduction of CD34+ cells with lymphomyeloid (2013) (0)
Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing (2022) (0)
Correction to: Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy (2022) (0)
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single-center report on 10 patients Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a (2011) (0)
GENE THERAPY OF THE β-HEMOGLOBINOPATHIES (2019) (0)
An Autosomal Dominant Form of Ras-Related C3 Botulinum Toxin Substrate 2 (RAC2) Is Associated with Haematopoiesis Failure (2021) (0)
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transplantation: a survey of the European experience, 1993-2002 Treatment of CD40 ligand deficiency by hematopoietic stem cell (2013) (0)
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Gene Therapy of Human Blnk Deficiency in NOD/SCID/Gc Ko Murine Model (2011) (0)
Treatment of Post- HSCT Immunodeficiency By Infusion of Ex Vivo- Generated T Cell Precursors from Adult and Cord Blood Hematopoietic Stem and Progenitor Cells (2015) (0)
Abstract session I–Transplantation (2004) (0)
Combinatorial RNA interference as a gene therapy strategy for HIV-1 infection (2013) (0)
Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia (2022) (0)
Correction of Artemis Deficiency In Murine Hematopoietic Stem Cell by I-Sce 1 Meganuclease and Artemis Recombination Matrix Mediated Homologous Recombination. (2010) (0)
Rapid and Safe T Cell Immune Reconstitution By T Cell Progenitor Injection Following Haploidentical Transplantation for Severe Combined Immunodeficiency (SCID) (2021) (0)
A ROLE FOR INO80, A CORE PROTEIN OF A CHROMATIN REMODELING COMPLEX, IN CLASS SWITCH RECOMBINATION (2012) (0)
231. Mixed Chimerism After Allogeneic Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: Preliminary Results on Peripheral Blood Sorted Subpopulations and Erythroid Progenitors (2016) (0)
Ex Vivo Production of Large Numbers of Genetically Modified NK Cells from Cord Blood or Mobilized Peripheral Blood CD34 + Cells Using Notch Ligand Delta-like 4 Culture System (2021) (0)
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice (2022) (0)
immunodeficiency single-center cohort of 90 patients with severe combined Long-term outcome after hematopoietic stem cell transplantation of a (2013) (0)
T-cell replete haploidentical hematopoietic stem cell transplantation (HSCT) as salvage therapy for acute rejection/graft failure in pediatric patients with nonmalignant disorders (2016) (0)
activity cells display hematopoietic - Lin + Human and murine amniotic fluid c-Kit (2014) (0)
The rationale for gene therapy in primary T cell immunodeficiencies (2013) (0)
225. Preserving CD4+ T-Cells Phenotype and Function Upon Ex Vivo Lentiviral Transduction (2016) (0)
PF441 RED BLOOD CELLS PROPERTIES IN PATIENTS WITH SICKLE CELL DISEASE TREATED WITH LENTIGLOBIN GENE THERAPY IN THE HGB-205 TRIAL (2019) (0)
Iconographies supplémentaires de l'article : Mammalian target of rapamycin inhibition counterbalances the inflammatory status of immune cells in patients with chronic granulomatous disease (2016) (0)
Assessment of Cardiac Iron Overload in Chonically Transfused Patients with Thalassemia, Sickle Cell Anemia, and Myelodysplastic Syndromes (2015) (0)
B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation (2015) (0)
SAFETY AND EFFICACY OF GENE THERAPY USING A MODIFIED SELF-INACTIVATING GAMMARETROVIRAL VECTOR FOR SCID-X1 (2017) (0)
Lymphoma and Chromosomal Instability in Artemis Deficient Mice - A Challenge for Long Term Malignancy Free Survival and Choice of Optimal Conditioning Regimen in Artemis Gene Therapy Trials. (2007) (0)
T-Cell Depleted Haematopoietic Stem Cells (HSC) Transplantation with Add Back of CD45RA Negative DLI: About 2 Cases (2012) (0)
20: GENE THERAPY OF THE BETA-HEMOGLOBINOPATHIES (2014) (0)
CLINICAL TRIALS AND OBSERVATIONS Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome (2016) (0)
Insertional mutagenesis and gene therapy (2005) (0)
Safety Following Autologous Transplantation with Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in the Northstar Hgb-204 Study (2018) (0)
The gene corrected clonal inventory in clinical gene therapy trials (2008) (0)
How Primary Immunodeficiencies Have Made Gene Therapy a Reality (2014) (0)
Anti CMV and/or Anti Adenovirus IFN-g-Positive CD4+ CD8+ T Lymphocytes for Treatment of Viral Infections After Allogeneic HSC Transplantation: First Results (2012) (0)
Human and murine amniotic ﬂuid c-Kit (cid:1) Lin (cid:2) cells display hematopoietic activity (2009) (0)
[Residual lymphocytes after specific depletion. Functional study]. (1992) (0)
Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency (2019) (0)
Anti CMV Immunotherapy with Donor Antigen Specific Lymphocytes Selected with GMP HLA Class 1 Tetramer Reagents. (2009) (0)
T-cell replete haploidentical bone marrow transplantation with serotherapy-containing conditioning and post-transplant cyclophosphamide for pediatric patients with non-malignant disorders (2016) (0)
SELF-INACTIVATING LENTIVIRAL VECTORS FOR CORRECTION OF RAG1 SEVERE COMBINED IMMUNODEFICIENCY (2012) (0)
Abnormalities of the Hematopoietic Stem Cell Compartment in Children After in Utero Exposure to AZT (2011) (0)
Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials (2022) (0)
HSC Transplantation for Hemoglobinopathies: Allogeneic or Autologous Gene-Modified HSC? (2011) (0)
A multimorphic mutation in IRF4 causes human autosomal dominant combined immunodeficiency (2023) (0)
134 HIV DNA Integration: Mechanism and Consequences (2012) (0)
Leukemia Prone B-Precursor Population in a p19ARF-/-RAG1-/- Mouse Model. (2009) (0)
Gene therapy for X-linked severe combined immunodeficiency and other hereditary diseases (2013) (0)
In Vitro Exposure to DL-4 Increases the in Vitro and In Vivo T-Lymphopoietic Potential of CB Derived CD34+ Progenitor Cells (2011) (0)
HSCT for children with severe combined immunodeficiencies (SCID) (2008) (0)
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiency. (2023) (0)
Therapeutic Plasma Exchange in Pediatrics for Immunologic Disorders; Tolerated and Safe Process for Pediatric Life-Threatening Conditions (2019) (0)
Omenn syndrome: more than a disorder of RAG1 or RAG2 genes (2005) (0)
Short title: Outcome of related and unrelated HSCT for SCID (2017) (0)
Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy. (2023) (0)
Promotion of mice cardiac allograft survival by transitory administration of LFA-1 antibody. 216 (1996) (0)
Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease (2016) (0)
In vitro expansion of murine amniotic fluid Lin - cKit high cells maintaining their hematopoietic potential (2010) (0)
SCIDs and related disorders (2015) (0)
559. Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin Locus (2016) (0)
a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients (2016) (0)
560. Generation of Functional Regulatory T Cells by FOXP3 Gene Transfer into CD4 T Cell from IPEX Patients (2016) (0)
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome (2022) (0)
Long-term outcome and lineage-speciﬁc chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study (2011) (0)
Vaccination and child mortality (2004) (0)
Bayesian Modeling Immune Reconstitution Apply to CD34+ Selected Stem Cell Transplantation for Severe Combined Immunodeficiency (2022) (0)
The human radiosensitive T-B-SCID define new gene(s) involved in V(D)J recombination and DNA repair (1997) (0)
Loss of p19Arf in a Rag1 (cid:1) / (cid:1) B-cell precursor population initiates acute B-lymphoblastic leukemia (2011) (0)
mTOR inhibition counterbalances the inflammatory status of immune cells in Chronic Granulomatous Disease. (2016) (0)
[Advances in gene therapy in genetic diseases of the hematopoietic system]. (2022) (0)
Gene therapy targeting hematopoietic stem cells: From the bench to the bedside (2008) (0)
Targeted Base Editing Strategies for Beta-Hemoglobinopathies (2022) (0)
Severe Combined Immunodeficiencies in Humans (1993) (0)
Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges (2019) (0)
Correction of SCID-XI by gene transfer: Current state and future plans. (2003) (0)
lymphoblastic leukemia B-cell precursor population initiates acute-/-in a Rag 1 p 19 Arf Loss of (2011) (0)
Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization (2018) (0)
Combinatorial RNA interference as a gene therapy strategy for HIV-1 infection (2013) (0)
425. Production of Gene Corrected T Cell Precursors for Therapy of SCID X1 (2016) (0)
From the Bone Marrow to the Thymic Niche (2014) (0)
Human adenylate kinase 2 deficiency causes a very profound haematopoietic defect associated with sensorineural deafness (Reticular Dysgenesis) (ESID Plenary Session VI: Novel Primary Immunodeficiencies and Late breakers) (2009) (0)
Further Characterization of T-Cellular Precursors Generated From CD34+ Progenitors by Exposure to Immobilized Notch Ligand Delta-Like 4 In Vitro. (2010) (0)
[Gene therapy of hereditary immunologic deficiencies]. (1996) (0)
[The bone marrow: a reserve of stem cells able to repair various tissues?]. (2004) (0)
[Prevention of intestinal allograft rejection by anti-adhesion molecule antibodies in a mouse model]. (1998) (0)
B Cells : A Single-Center Study of 22 Patients Transplantation in Patients With Severe Combined Immunodeficiency With Long-Term Chimerism and B-Cell Function After Bone Marrow (1999) (0)
Human Adenylate Kinase 2 Deficiency Inhibits Hematopoietic Cell Differentiation towards Neutrophil and T Lymphoid Lineages. (2009) (0)
Long-term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed-up to 7 Years after Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy (GT) and Factors Impacting Neutrophil and Platelet Engraftment (2023) (0)
Efficacy and Safety of Betibeglogene Autotemcel (beti-cel) Gene Therapy in 63 Patients with Transfusion-Dependent β-Thalassemia (TDT): 7-Year Post-Infusion Follow-up of Phase 1/2 and Phase 3 Studies (2022) (0)
Gene therapy of primary immunodeficiencies. (1998) (0)
Inflammation and inflammatory bowel disease-Blockade of the integrin aLb2 but not of integrins a4 an (2000) (0)
Selective allodepletion. (2005) (0)
O IX.3 Human SCIDs with increased sensitivity to ionizing radiations and impaired V(D)J rearrangements define new genes involved in DNA recombination/repair (1997) (0)
Les plates-formes de recherche sur les biothérapies (2007) (0)
Thérapie génique des déficits immunitaires héréditaires (1996) (0)
Conclusion – La révolution de la « smart » médecine (2011) (0)
Guérir un déficit immunitaire congénital. (2007) (0)
Une histoire du vivant et du bio mé dicament (2011) (0)
Thérapie génique des déficits immunitaires congénitaux : Thérapie Génique (2001) (0)
Prévention du rejet d'allogreffe intestinale par des anticorps anti-LFA-1 et anti-α4 chez la souris (1998) (0)
Produire le vivant. Un biomédicament pas à pas (2011) (0)
Introduction – Biomédicament : des biotechnologies aux médicaments de l'avenir (2011) (0)
O3-2 Étude comparative de trois méthodes physiques de séparation cellulaire et résultats de leur application clinique (1998) (0)
P3-16 Reconstitution hématologique après autogreffe de cellules souches périphériques selon la quantité de CFU-GM mesurée après décongélation (1998) (0)
20 ans de thérapie génique (2009) (0)
Le biomédicament pour la médecine de demain (2011) (0)
Déficit de l’immunité antivirale : EBV, CMV, adénovirus (1997) (0)
Approches experimentales de la prevention du rejet de greffet medullaire incompatible (1993) (0)
Thérapie génique du déficit combiné sévère lié à l'X Efficacité (2004) (0)
O3-8 Étude de la conservation du potentiel lymphoïde après transfert de gène dans les cellules hématopotétiques humaines CD34+ (1998) (0)
Thérapie génique des déficits immunitaires congénitaux (2002) (0)
Le biomédicament, pour une définition commune (2011) (0)
Ils vont bien… un an après (2002) (0)
Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients. (2023) (0)

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