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List of works by Marina Cavazzana

20 years of gene therapy for SCID.

scientific article

A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells

scientific article published on 08 August 2018

A double-blind low dose-finding phase II study of granulocyte colony-stimulating factor combined with chemotherapy for stem cell mobilization in patients with non-Hodgkin's lymphoma.

scientific article published in April 2006

A human immunodeficiency caused by mutations in the PIK3R1 gene

scientific article

A human immunodeficiency caused by mutations in the PIK3R1 gene

article

A human postnatal lymphoid progenitor capable of circulating and seeding the thymus

scientific article

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

scientific article

A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome

scientific article published on May 2007

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

scientific article

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

scientific article

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

scientific article published on 01 April 1996

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

scientific article

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

scientific article published in January 2003

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.

scientific article

AK2 deficiency compromises the mitochondrial energy metabolism required for differentiation of human neutrophil and lymphoid lineages

scientific article

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

scientific article published on 7 September 2017

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

scientific article published in May 2002

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

scientific article published on July 2010

Allogeneic bone marrow transplantation in mevalonic aciduria

scientific article

Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency

scientific article (publication date: March 2009)

Amniotic Fluid Stem Cells Restore the Muscle Cell Niche in a HSA-Cre, SmnF7/F7 Mouse Model

scientific article published on 01 August 2012

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

scientific article published on 04 August 2018

An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, and MPN domains 1 (MYSM1) in human hematopoiesis and lymphocyte differentiation

scientific article published on 25 July 2015

An inherited immunoglobulin class-switch recombination deficiency associated with a defect in the INO80 chromatin remodeling complex

scientific article

Artemis, a novel DNA double-strand break repair/V(D)J recombination protein, is mutated in human severe combined immune deficiency.

scientific article

Arterio-venous fistula for automated red blood cells exchange in patients with sickle cell disease: Complications and outcomes.

scientific article

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

scientific article published on 01 January 1994

Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients

scientific article

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

scientific article published on 01 February 2019

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

scientific article published on 4 November 2022

Basic research tries to decrease the risks of translational medicine

scientific article

Bone marrow failure in Fanconi anemia is triggered by an exacerbated p53/p21 DNA damage response that impairs hematopoietic stem and progenitor cells

scientific article

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

scientific article published on 01 February 1994

Bone marrow transplantation attenuates the myopathic phenotype of a muscular mouse model of spinal muscular atrophy

scientific article published on 3 August 2006

Bone marrow transplantation for primary immunodeficiency diseases

scientific article published on February 2010

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

scientific article published on 01 September 1996

Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center

scientific article

Bone marrow transplantation in major histocompatibility complex class II deficiency: a single-center study of 19 patients

scientific article published on January 1995

Bone marrow-derived mononuclear cell therapy induces distal angiogenesis after local injection in critical leg ischemia

scientific article

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

scientific article published on 10 October 2006

CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: A preliminary study

scientific article published on 3 October 2014

Cardiac iron overload in chronically transfused patients with thalassemia, sickle cell anemia, or myelodysplastic syndrome

scientific article

Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

scientific article

Characteristics of antigen-independent and antigen-dependent interaction of dendritic cells with CD4+ T cells

scientific article published on 01 August 1995

Characteristics of antigen-independent and antigen-dependent interaction of dendritic cells with CD4+ T cells

scientific article published on 01 January 1995

Characterization of antigen-specific repertoire diversity following in vitro restimulation by a recombinant adenovirus expressing human cytomegalovirus pp65.

scientific article published on March 2003

Circulating endothelial cells as markers of endothelial dysfunction during hematopoietic stem cell transplantation for pediatric primary immunodeficiency

scholarly article by Fabien Touzot et al published November 2014 in The Journal of Allergy and Clinical Immunology

Clinical and immunologic outcome of patients with cartilage hair hypoplasia after hematopoietic stem cell transplantation.

scientific article

Clinical and immunologic phenotype associated with activated phosphoinositide 3-kinase δ syndrome 2: A cohort study ⬇️

scientific article published on 21 April 2016

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

scientific article published on 7 December 2004

Combinatorial RNA interference as a gene therapy strategy for HIV-1 infection.

scientific article published on 19 September 2013

Comparing genotoxic signatures in cord blood cells from neonates exposed in utero to zidovudine or tenofovir

scientific article published in July 2015

Comparison of Lenograstim vs Filgrastim Administration following Chemotherapy for Peripheral Blood Stem Cell (PBSC) Collection: A Retrospective Study of 126 Patients

Competition within the early B-cell compartment conditions B-cell reconstitution after hematopoietic stem cell transplantation in nonirradiated recipients

article

Concise Review: Boosting T-Cell Reconstitution Following Allogeneic Transplantation-Current Concepts and Future Perspectives

scientific article published on 18 March 2019

Correction of Fas (CD95) deficiency by haploidentical bone marrow transplantation.

scientific article

Correction of genetic blood defects by gene transfer.

scientific article

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

scientific article published on 27 May 2011

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

scientific article published on 3 May 2012

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

scientific article published on 29 April 2011

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

scientific article published on 14 April 2008

Depletion of alloreactive T cells by a specific anti-interleukin-2 receptor p55 chain immunotoxin does not impair in vitro antileukemia and antiviral activity.

scientific article published on May 1999

Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach

scientific article published on 19 April 2016

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. ⬇️

scientific article published on 16 March 2018

Dix ans de thérapie génique Réflexions

scientific article published on 01 February 2010

Does haploidentical transplantation in children with primary immunodeficiencies have the potential to exploit donor NK cell alloreactivity?

scientific article published in December 2004

Donor T lymphocyte infusion following ex vivo depletion of donor anti-host reactivity by a specific anti–interleukin-2 receptor P55 chain immunotoxin

scientific article published on 01 November 2002

Donor-targeted serotherapy as a rescue therapy for steroid-resistant acute GVHD after HLA-mismatched kidney transplantation

scientific article published on 17 February 2020

Dynamics of Thymus-Colonizing Cells during Human Development

article

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

scientific article

Déficit de l’immunité antivirale : EBV, CMV, adénovirus

scientific article published on 01 October 1997

Early posttransplantation donor-derived invariant natural killer T-cell recovery predicts the occurrence of acute graft-versus-host disease and overall survival

scientific article published on 22 June 2012

Early-onset hypogammaglobulinemia: A survey of 44 patients

scientific article published on 08 May 2015

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

scientific article published on 12 February 2020

Efficacité et sécurité du traitement par thérapie génique des patients atteints du syndrome de Wiskott-Aldrich

scientific article published on 16 December 2015

Efficacy of gene therapy for SCID is being confirmed

scientific article published in December 2004

Efficacy of gene therapy for X-linked severe combined immunodeficiency

scientific article

Evaluation of an algorithm based on peripheral blood hematopoietic progenitor cell and CD34+ cell concentrations to optimize peripheral blood progenitor cell collection by apheresis

scientific article published in October 2007

Evaluation of antithymocyte globulin pharmacokinetics and pharmacodynamics in children

scholarly article by Naïm Bouazza et al published January 2016 in The Journal of Allergy and Clinical Immunology

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

scientific article published on 01 August 2019

Failure of SCID-X1 gene therapy in older patients

scientific article

Failure of bone marrow transplantation to eradicate HIV reservoir despite efficient HAART

article

Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks

scientific article

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

scientific article

First experience of autologous peripheral blood stem cell mobilization with biosimilar granulocyte colony- stimulating factor

article by François Lefrère et al published 10 March 2011 in Advances in Therapy

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

scientific article

Functional study of residual T lymphocytes after specific elimination of alloreactive T cells by a specific anti-interleukin-2 receptor Bk chain immunotoxin

scientific article published in December 1993

Gene Therapy Approaches to Hemoglobinopathies.

scientific article published in October 2017

Gene Therapy for Hemoglobinopathies

article

Gene Therapy for Primary Immunodeficiencies

article

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

scientific article

Gene Therapy for β-Hemoglobinopathies

scientific article

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

article

Gene Therapy in a Patient with Sickle Cell Disease

scientific article published on March 2017

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene

scientific article published on February 2016

Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View.

scientific article

Gene therapy for Wiskott-Aldrich Syndrome.

scientific article

Gene therapy for human severe combined immunodeficiencies

scientific article

Gene therapy for human severe combined immunodeficiencies

article

Gene therapy for immunodeficiency diseases

scientific article published in October 2004

Gene therapy for inherited immunodeficiency

scientific article

Gene therapy for primary adaptive immune deficiencies

scientific article published on June 2011

Gene therapy for primary immunodeficiencies

scientific article published on May 2010

Gene therapy for primary immunodeficiencies.

scientific article

Gene therapy for primary immunodeficiencies: Part 1.

scientific article published on 12 September 2012

Gene therapy for primary immunodeficiencies: Part 2.

scientific article published on 18 August 2012

Gene therapy for severe combined immunodeficiency

scientific article

Gene therapy for severe combined immunodeficiency: are we there yet?

scientific article

Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency

scientific article published on 22 August 2002

Gene therapy of X-linked severe combined immunodeficiency

scientific article

Gene therapy of X-linked severe combined immunodeficiency

article

Gene therapy of X-linked severe combined immunodeficiency

scientific article published on 01 December 2002

Gene therapy of hemoglobinopathies: progress and future challenges

scientific article published on 01 October 2019

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

scientific article (publication date: 28 April 2000)

Gene therapy of inherited diseases

scientific article published on June 2008

Gene therapy of metabolic diseases

scientific article published on April 2006

Gene therapy of primary T cell immunodeficiencies

scientific article published on 10 April 2013

Gene therapy of primary immunodeficiencies

scientific article

Gene therapy of severe combined immunodeficiencies

article

Gene therapy of severe combined immunodeficiencies

scientific article published on 01 May 2001

Gene therapy of severe combined immunodeficiencies

scientific article published on 01 January 2001

Gene therapy of severe combined immunodeficiencies.

scientific article

Gene therapy of severe combined immunodeficiencies: from mice to humans.

scientific article published in August 2002

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

scientific article published on February 2010

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

scientific article published on 01 June 2019

Gene therapy: X-SCID transgene leukaemogenicity

scientific article

Gene transfer for activation of CMV specific T cells

scientific article published on May 2004

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency

scientific article published on 21 December 2017

Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice.

scientific article published on 31 October 2016

Generation of adult human T-cell progenitors for immunotherapeutic applications

scientific article published on 2 December 2017

Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination

scientific article published on 4 April 2013

HIV-1 Nef protein expression in human CD34+ progenitors impairs the differentiation of an early T/NK cell precursor

article

Haematopoietic stem cell transplantation for SCID patients: where do we stand?

scientific article

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

scientific article

Hematopoietic stem cell gene therapy: progress on the clinical front

scientific article published on 28 February 2014

Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-SCETIDE-PIDTC Study

scientific article published on 17 January 2019

Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients

scientific article published on 29 April 2009

Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients

scientific article

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

scientific article

Hepatitis E virus in hematopoietic stem cell donors: Towards a systematic HEV screening of donors?

scientific article

High-dose CD34+ cells are not clinically relevant in reducing cytopenia and blood component consumption following myeloablative therapy and peripheral blood progenitor cell transplantation as compared with standard dose

scientific article published on 01 April 2002

Human T-lymphoid progenitors generated in a feeder-cell-free Delta-like-4 culture system promote T-cell reconstitution in NOD/SCID/γc(-/-) mice

scientific article published on August 2012

Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness

scientific article

Human and murine amniotic fluid c-Kit+Lin- cells display hematopoietic activity

scientific article published on 12 February 2009

Human hematopoiesis: from CD34 cells to T lymphocytes

scientific article published in February 2007

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

Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions

scientific article

IL-7 effect on immunological reconstitution after HSCT depends on MHC incompatibility

scientific article published on 01 September 2004

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

scientific article

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions.

scientific article published on 18 December 2016

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

scientific article published on 18 September 2009

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

scientific article published in The Lancet

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

article

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

scientific article

In vivo infusion of anti-LFA-1 and anti-CD2 antibodies prevents graft failure after HLA partially incompatible bone marrow transplantation in children with high risk acute lymphoblastic leukaemia

scientific article published on December 1997

Increased radiosensitivity of granulocyte macrophage colony-forming units and skin fibroblasts in human autosomal recessive severe combined immunodeficiency

scientific article

Induction of NKG2D ligands by gamma radiation and tumor necrosis factor-alpha may participate in the tissue damage during acute graft-versus-host disease

scientific article

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

scientific article published on 8 March 2018

Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell–depleted bone marrow transplantationA retrospective European survey from the European Group for Bone Marrow Transplantation and the European Society

scientific article published on 01 June 1999

Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies

scientific article published on 24 March 2017

Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation

scientific article published on 03 May 2019

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

scientific article published on 23 August 2011

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

scientific article

Integration of retroviruses: a fine balance between efficiency and danger

scientific article

Intensive and prolonged treatment of focal and segmental glomerulosclerosis recurrence in adult kidney transplant recipients: a pilot study.

scientific article published on 16 March 2009

International AIDS Society global scientific strategy: towards an HIV cure 2016.

scientific article published on 11 July 2016

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

scientific article

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

scientific article (publication date: 17 October 2003)

Lack of detectable defect in DNA double-strand break repair and DNA-dependent protein kinase activity in radiosensitive human severe combined immunodeficiency fibroblasts

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

scientific article published in January 2012

Les ligands Delta et la niche du développement lymphoïde

scientific article published on 01 January 2007

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency

scientific article published on 10 December 2016

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

scientific article published on 01 February 2007

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

scientific article published in October 1999

Long-term follow-up of patients who received transplants for aplastic anemia or leukemia

scientific article published on 01 February 1987

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.

scientific article

Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity

scientific article

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

scientific article published on 23 January 2009

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

scientific article

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

scientific article published on 27 September 2007

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

scientific article published on 24 January 2022

Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99

scientific article published in The Lancet

Loss of ARHGEF1 causes a human primary antibody deficiency

scientific article published on 04 February 2019

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

scientific article

Lymphoid-affiliated genes are associated with active histone modifications in human hematopoietic stem cells

scientific article published on 14 July 2008

Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression

scientific article published on 30 March 2010

Lymphopoiesis in transgenic mice over-expressing Artemis

scientific article

Mammalian target of rapamycin inhibition counterbalances the inflammatory status of immune cells in patients with chronic granulomatous disease

scientific article

Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).

scientific article published on 14 February 2007

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

article

Medical perspectives of adults and embryonic stem cells

scientific article published on 01 October 2002

Mesenchymal stromal cells can be derived from bone marrow CD133+ cells: implications for therapy

scientific article

Mutations in the adaptor-binding domain and associated linker region of p110δ cause Activated PI3K-δ Syndrome 1 (APDS1).

scientific article published on 20 April 2017

Naturally occurring primary deficiencies of the immune system

scientific article published on January 1997

New ISSCR guidelines underscore major principles for responsible translational stem cell research

scientific article published on December 2008

Occurrence of myelodysplastic syndrome in 2 patients with reticular dysgenesis

scientific article published on 31 March 2011

Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells

scientific article published in February 2001

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.

scientific article published on 12 July 2012

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

scientific article published on April 2015

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

scientific article

Partial T and B lymphocyte immunodeficiency and predisposition to lymphoma in patients with hypomorphic mutations in Artemis

scientific article

Partial T and B lymphocyte immunodeficiency and predisposition to lymphoma in patients with hypomorphic mutations in Artemis

article by Despina Moshous et al published 1 February 2003 in Journal of Clinical Investigation

Peripheral blood stem cell and gene therapy

article

Plerixafor enables the safe, rapid, efficient mobilization of haematopoietic stem cells in sickle cell disease patients after exchange transfusion

scientific article published on 22 February 2018

Pre-transplant donor CD4− invariant NKT cell expansion capacity predicts the occurrence of acute graft-versus-host disease

scientific article published on 14 October 2016

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

scientific article

Prevention of EBV-induced B-lymphoproliferative disorder by ex vivo marrow B-cell depletion in HLA-phenoidentical or non-identical T-depleted bone marrow transplantation

scientific article published on 01 November 1998

Prevention of bone marrow and cardiac graft rejection in an H-2 haplotype disparate mouse combination by an anti-LFA-1 antibody

scientific article published on 01 June 1995

Primary microcephaly, impaired DNA replication, and genomic instability caused by compound heterozygous ATR mutations

scientific article

Prévention du rejet d'allogreffe intestinale par des anticorps antimolécules d'adhésion dans un modèle murin

scientific article published on 01 April 1998

Real-time definition of non-randomness in the distribution of genomic events

scientific article

Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

scientific article published on 9 December 2013

Residual lymphocytes after specific depletion. Functional study

scientific article published in December 1992

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

scientific article published on 27 November 2007

Results of allogeneic bone marrow transplantation in patients with leukocyte adhesion deficiency

scientific article published on 01 August 1995

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

scientific article

Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome

scientific article

Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease

scientific article published on 24 April 2019

Role of calcineurin-dependent drugs on the immunosuppressive effect induced by the anti-LFA-1 antibody in a fetal intestinal transplantation model in mice

scientific article (publication date: November 1999)

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

SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID.

scientific article published on 21 October 2013

Safety of CD34 Hematopoietic Stem Cells and CD4 T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma

article

Safety of hematopoietic stem cell transplantation from hepatitis B core antibodies-positive donors with low/undetectable viremia in HBV-naïve children.

scientific article published on 28 September 2013

Second allogeneic haematopoietic stem cell transplantation in relapsed acute and chronic leukaemias for patients who underwent a first allogeneic bone marrow transplantation: a survey of the Societe Francaise de Greffe de Moelle (SFGM)

scientific article published on 01 February 2000

Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies

scientific article published on 28 October 2020

Severe combined immunodeficiencies and related disorders

scientific article

Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV.

scientific article published on January 2006

Severe combined immunodeficiency caused by deficiency in either the delta or the epsilon subunit of CD3

scientific article

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

scientific article

Severe cutaneous papillomavirus disease after haemopoietic stem-cell transplantation in patients with severe combined immune deficiency caused by common gammac cytokine receptor subunit or JAK-3 deficiency

scientific article published in June 2004

Shortening the immunodeficient period after hematopoietic stem cell transplantation

scientific article

Sickle SCAN™ (BioMedomics) fulfills analytical conditions for neonatal screening of sickle cell disease

scientific article published on 01 August 2018

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

scientific article published on 13 December 2016

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

scientific article

Specific elimination of alloreactive T cells by an anti-interleukin-2 receptor B chain-specific immunotoxin

scientific article

Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells.

scientific article published on 17 June 2008

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

scientific article

Stem cell transplantation for immunodeficiency

scientific article published on 01 January 1998

Stem cell transplantation for primary immunodeficiencies: the European experience

scientific article

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

scientific article published in January 2012

Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.

scientific article published on 26 June 2014

Successful haematopoietic stem cell transplantation in a case of pulmonary alveolar proteinosis due to GM-CSF receptor deficiency

scientific article published on 20 December 2017

Successful in utero stem cell transplantation in X-linked severe combined immunodeficiency

scientific article published on 01 February 2019

Successful peripheral blood stem cell harvesting with granulocyte colony-stimulating factor alone after previous mobilization failure

scientific article published on 01 December 2004

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

scientific article (publication date: 18 April 2002)

Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia: Analysis of cost-effectiveness based on French real field data

scientific article

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

article

T-cell-depleted HLA non-identical bone marrow transplantation in the child: prevention of graft-versus-host reaction by administration of donor T lymphocytes alloreactive against the recipient

scientific article

The BLNK adaptor protein has a nonredundant role in human B-cell differentiation

scientific article published on 28 February 2014

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

article

The biology of allogeneic hematopoietic cell resistance

scientific article

The dose of granulocyte-colony-stimulating factor after chemopriming treatment does not influence apheresis yield of progenitor cells: a retrospective study of 91 cases

scientific article published on 01 November 1999

The feasibility of peripheral blood stem cell collection for autograft following failure in bone marrow aspiration

scientific article published on 01 June 1998

The future of gene therapy

scientific article

The timing of granulocyte-colony-stimulating factor administration after chemotherapy does not affect stem and progenitor cell apheresis yield: a retrospective study of 65 cases

scientific article published on 01 June 1999

Thérapie génique des déficits immunitaires héréditaires

scientific article published on 01 January 1996

Thérapie génique du déficit immunitaire combiné sévère lié à l'X (DICS-X1)

scientific article published on 01 February 2001

Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.

scientific article published in March 2003

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

scientific article

Transfusion-related adverse events are decreased in pregnant women with sickle cell disease by a change in policy from systematic transfusion to prophylactic oxygen therapy at home: A retrospective survey by the international sickle cell disease obs

scientific article published on 30 March 2018

Transient familial haemophagocytic lymphohistiocytosis reactivation post-CD34 haematopoietic stem cell transplantation

scientific article

Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced dysfunction

scientific article published on 08 November 2021

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

scientific article

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

scientific article published on 31 July 2010

Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002

scientific article published on 02 October 2003

Treatment of Omenn syndrome by bone marrow transplantation

scientific article published on 01 July 1995

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

scientific article

Treatment of familial hemophagocytic lymphohistiocytosis with bone marrow transplantation from HLA genetically nonidentical donors

scientific article published on 01 December 1997

Treatment of juvenile chronic myelomonocytic leukemia by allogeneic bone marrow transplantation

scientific article published on 01 June 1994

Treatment of the Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome (IPEX) by Allogeneic Bone Marrow Transplantation

article

Trisomy 21 mid-trimester amniotic fluid induced pluripotent stem cells maintain genetic signatures during reprogramming: implications for disease modeling and cryobanking

scientific article published on 27 August 2014

Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells.

scientific article published on 13 March 2014

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

scientific article published in August 2007

X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease

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X-linked primary immunodeficiency associated with hemizygous mutations in the moesin (MSN) gene

scientific journal article

[Cell therapy for inherited diseases of the hematopoietic system]

scientific article published on 15 May 2007

[Curing a congenital immunologic deficiency]

scientific article published on 01 October 2007

[Evidence for the widespread use of neonatal screening for sickle cell disease]

scientific article published on 16 April 2018

[Gene therapy for immune deficiencies]

scientific article published on 01 January 2000

[Gene therapy for sickle cell disease]

scientific article published on 01 May 2017

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

scientific article published on 01 January 2004

[Gene therapy of severe combined immunodeficiencies]

scientific article published on 01 June 2000

[Gene therapy: many more questions than answers]

scientific article published on 01 May 2017

[Gene therapy: where do we stand?].

scientific article published in April 2013

gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function.

scientific article published on April 1996

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

scientific article published on December 1998

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