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Lista de obras de Marina Cavazzana

20 years of gene therapy for SCID.

artículo científico publicado en 2010

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

artículo científico publicado en 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.

artículo científico publicado en 2006

A human immunodeficiency caused by mutations in the PIK3R1 gene

artículo científico publicado en 2014

A human immunodeficiency caused by mutations in the PIK3R1 gene

article

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

artículo científico publicado en 2007

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

artículo científico publicado en 1998

A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome

artículo científico publicado en 2007

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

artículo científico publicado en 2011

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

artículo científico publicado en 2014

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

artículo científico publicado en 1996

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

artículo científico publicado en 2010

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

artículo científico publicado en 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.

artículo científico publicado en 2012

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

artículo científico publicado en 2015

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

artículo científico publicado en 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

artículo científico publicado en 2002

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

artículo científico publicado en 2010

Allogeneic bone marrow transplantation in mevalonic aciduria

artículo científico publicado en 2007

Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency

artículo científico publicado en 2009

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

artículo científico publicado en 2012

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

artículo científico publicado en 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

artículo científico publicado en 2015

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

artículo científico publicado en 2014

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

artículo científico publicado en 2001

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

artículo científico publicado en 2016

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

artículo científico publicado en 1994

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

artículo científico publicado en 2003

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

artículo científico publicado en 2019

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

artículo científico publicado en 2022

Basic research tries to decrease the risks of translational medicine

artículo científico publicado en 2009

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

artículo científico publicado en 2012

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

artículo científico publicado en 1994

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

artículo científico publicado en 2006

Bone marrow transplantation for primary immunodeficiency diseases

artículo científico publicado en 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

artículo científico publicado en 1996

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

artículo científico publicado en 1996

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

artículo científico publicado en 1995

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

artículo científico publicado en 2008

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

artículo científico publicado en 2006

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

artículo científico publicado en 2014

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

artículo científico publicado en 2017

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

artículo científico publicado en 2012

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

artículo científico publicado en 1995

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

artículo científico publicado en 1995

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

artículo científico publicado en 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.

artículo científico publicado en 2010

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

artículo científico publicado en 2016

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

artículo científico publicado en 2004

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

artículo científico publicado en 2013

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

artículo científico publicado en 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

artículo científico publicado en 2019

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

artículo científico publicado en 1997

Correction of genetic blood defects by gene transfer.

artículo científico publicado en 2001

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

artículo científico publicado en 2011

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

artículo científico publicado en 2012

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

artículo científico publicado en 2011

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

artículo científico publicado en 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.

artículo científico publicado en 1999

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

artículo científico publicado en 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. ⬇️

artículo científico publicado en 2018

Dix ans de thérapie génique Réflexions

artículo científico publicado en 2010

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

artículo científico publicado en 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

artículo científico publicado en 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

artículo científico publicado en 2010

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

artículo científico publicado en 1997

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

artículo científico publicado en 2012

Early-onset hypogammaglobulinemia: A survey of 44 patients

artículo científico publicado en 2015

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

artículo científico publicado en 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

artículo científico publicado en 2004

Efficacy of gene therapy for X-linked severe combined immunodeficiency

artículo científico publicado en 2010

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

artículo científico publicado en 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

artículo científico publicado en 2019

Failure of SCID-X1 gene therapy in older patients

artículo científico publicado en 2005

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

artículo científico publicado en 2017

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

artículo científico publicado en 2015

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

artículo científico publicado en 2006

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

artículo científico publicado en 1993

Gene Therapy Approaches to Hemoglobinopathies.

artículo científico publicado en 2017

Gene Therapy for Hemoglobinopathies

article

Gene Therapy for Primary Immunodeficiencies

article

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

artículo científico publicado en 2016

Gene Therapy for β-Hemoglobinopathies

artículo científico publicado en 2017

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

article

Gene Therapy in a Patient with Sickle Cell Disease

artículo científico publicado en 2017

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

artículo científico publicado en 2016

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

artículo científico publicado en 2016

Gene therapy for Wiskott-Aldrich Syndrome.

artículo científico publicado en 2014

Gene therapy for human severe combined immunodeficiencies

artículo científico publicado en 2001

Gene therapy for human severe combined immunodeficiencies

article

Gene therapy for immunodeficiency diseases

artículo científico publicado en 2004

Gene therapy for inherited immunodeficiency

artículo científico publicado en 2014

Gene therapy for primary adaptive immune deficiencies

artículo científico publicado en 2011

Gene therapy for primary immunodeficiencies

artículo científico publicado en 2010

Gene therapy for primary immunodeficiencies.

artículo científico publicado en 2015

Gene therapy for primary immunodeficiencies: Part 1.

artículo científico

Gene therapy for primary immunodeficiencies: Part 2.

artículo científico publicado en 2012

Gene therapy for severe combined immunodeficiency

artículo científico publicado en 2005

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

artículo científico publicado en 2007

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

artículo científico publicado en 2002

Gene therapy of X-linked severe combined immunodeficiency

artículo científico publicado en 2002

Gene therapy of X-linked severe combined immunodeficiency

article

Gene therapy of X-linked severe combined immunodeficiency

artículo científico publicado en 2002

Gene therapy of hemoglobinopathies: progress and future challenges

artículo científico publicado en 2019

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

artículo científico publicado en 2000

Gene therapy of inherited diseases

artículo científico publicado en 2008

Gene therapy of metabolic diseases

artículo científico publicado en 2006

Gene therapy of primary T cell immunodeficiencies

artículo científico

Gene therapy of primary immunodeficiencies

artículo científico publicado en 1997

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

artículo científico publicado en 2001

Gene therapy of severe combined immunodeficiencies.

artículo científico publicado en 2000

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

artículo científico publicado en 2002

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

artículo científico publicado en 2010

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

artículo científico publicado en 2019

Gene therapy: X-SCID transgene leukaemogenicity

artículo científico publicado en 2006

Gene transfer for activation of CMV specific T cells

artículo científico publicado en 2004

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

artículo científico publicado en 2017

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

artículo científico publicado en 2016

Generation of adult human T-cell progenitors for immunotherapeutic applications

artículo científico publicado en 2017

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

artículo científico publicado en 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?

artículo científico publicado en 2012

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

artículo científico publicado en 2009

Hematopoietic stem cell gene therapy: progress on the clinical front

artículo científico publicado en 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

artículo científico publicado en 2009

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

artículo científico publicado en 2006

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

artículo científico publicado en 2014

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

artículo científico publicado en 2015

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

artículo científico publicado en 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

artículo científico publicado en 2012

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

artículo científico publicado en 2009

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

artículo científico publicado en 2009

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

artículo científico publicado en 2016

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

artículo científico publicado en 2016

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

artículo científico publicado en 2016

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

artículo científico publicado en 2009

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

artículo científico publicado en 2002

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

artículo científico publicado en 2002

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

artículo científico publicado en 1997

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

artículo científico publicado en 1993

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

artículo científico publicado en 2008

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

artículo científico publicado en 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

artículo científico publicado en 1999

Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies

artículo científico publicado en 2017

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

artículo científico publicado en 2019

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

artículo científico publicado en 2011

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

artículo científico publicado en 2008

Integration of retroviruses: a fine balance between efficiency and danger

artículo científico publicado en 2005

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

artículo científico publicado en 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?

artículo científico publicado en 2011

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

artículo científico publicado en 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

artículo científico publicado en 2012

Les ligands Delta et la niche du développement lymphoïde

artículo científico publicado en 2007

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

artículo científico publicado en 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.

artículo científico publicado en 1999

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

artículo científico publicado en 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.

artículo científico publicado en 1998

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

artículo científico publicado en 2005

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

artículo científico publicado en 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

artículo científico publicado en 2011

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

artículo científico publicado en 2007

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

artículo científico publicado en 2022

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

artículo científico publicado en 2003

Loss of ARHGEF1 causes a human primary antibody deficiency

artículo científico publicado en 2019

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

artículo científico publicado en 2011

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

artículo científico publicado en 2008

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

artículo científico publicado en 2010

Lymphopoiesis in transgenic mice over-expressing Artemis

artículo científico publicado en 2015

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

artículo científico publicado en 2016

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

artículo científico publicado en 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

artículo científico publicado en 2009

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

artículo científico publicado en 2017

Naturally occurring primary deficiencies of the immune system

artículo científico publicado en 1997

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

artículo científico publicado en 2008

Occurrence of myelodysplastic syndrome in 2 patients with reticular dysgenesis

artículo científico publicado en 2011

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

artículo científico publicado en 2001

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

artículo científico publicado en 2012

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

artículo científico publicado en 2015

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

artículo científico publicado en 2013

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

artículo científico publicado en 2003

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

artículo científico publicado en 2018

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

artículo científico publicado en 2016

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

artículo científico publicado en 2015

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

artículo científico publicado en 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

artículo científico publicado en 2012

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

artículo científico publicado en 1998

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

artículo científico publicado en 2007

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

artículo científico publicado en 2013

Residual lymphocytes after specific depletion. Functional study

artículo científico publicado en 1992

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

artículo científico publicado en 2007

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

artículo científico publicado en 1995

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

artículo científico publicado en 2017

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

artículo científico publicado en 2009

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

artículo científico publicado en 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.

artículo científico publicado en 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.

artículo científico publicado en 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)

artículo científico publicado en 2000

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

artículo científico publicado en 2020

Severe combined immunodeficiencies and related disorders

artículo científico publicado en 2015

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

artículo científico publicado en 2006

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

artículo científico publicado en 2004

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

artículo científico publicado en 2005

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

artículo científico publicado en 2004

Shortening the immunodeficient period after hematopoietic stem cell transplantation

artículo científico publicado en 2009

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

artículo científico publicado en 2018

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

artículo científico publicado en 2016

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

artículo científico publicado en 2016

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

artículo científico publicado en 1990

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

artículo científico publicado en 2008

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

artículo científico publicado en 2011

Stem cell transplantation for immunodeficiency

artículo científico publicado en 1998

Stem cell transplantation for primary immunodeficiencies: the European experience

artículo científico publicado en 2014

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

artículo científico publicado en 2012

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

artículo científico publicado en 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

artículo científico publicado en 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

artículo científico publicado en 2002

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

artículo científico publicado en 2015

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

artículo científico publicado en 2001

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

artículo científico publicado en 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

artículo científico publicado en 2009

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

artículo científico publicado en 2004

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

artículo científico publicado en 1999

Thérapie génique des déficits immunitaires héréditaires

artículo científico publicado en 1996

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

artículo científico publicado en 2001

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

artículo científico publicado en 2003

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

artículo científico publicado en 2010

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

artículo científico publicado en 2018

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

artículo científico publicado en 2005

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

artículo científico publicado en 2021

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

artículo científico publicado en 2012

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

artículo científico publicado en 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

artículo científico publicado en 1995

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

artículo científico publicado en 2005

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

artículo científico publicado en 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

artículo científico publicado en 2014

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

artículo científico publicado en 2014

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

artículo científico publicado en 2007

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

artículo científico publicado en 2010

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]

artículo científico publicado en 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]

artículo científico publicado en 2018

[Gene therapy for immune deficiencies]

artículo científico publicado en 2000

[Gene therapy for sickle cell disease]

artículo científico publicado en 2017

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

artículo científico publicado en 2004

[Gene therapy of severe combined immunodeficiencies]

scientific article published on 01 June 2000

[Gene therapy: many more questions than answers]

artículo científico publicado en 2017

[Gene therapy: where do we stand?].

artículo científico publicado en 2013

[Human hematopoiesis: from CD34 cells to T lymphocytes].

artículo científico publicado en 2007

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

artículo científico publicado en 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

artículo científico publicado en 1998

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