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List of works by Adrian Thrasher

"Darwinian" tumor-suppression model unsupported in clinical experience

scientific article published on September 2014

"Special exemptions": should they be put on trial?

scientific article published in February 2013

A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).

scientific article published on 18 January 2017

A Personal Reflection from London

scientific article published in November 2017

A congenital activating mutant of WASp causes altered plasma membrane topography and adhesion under flow in lymphocytes

scientific article

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

scientific article

A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity

scientific article published on 31 March 2009

A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells

scientific article published on 15 December 2005

A phagocyte dilemma.. ⬇️

scientific article published on March 1, 2011

A robust model for read count data in exome sequencing experiments and implications for copy number variant calling

scientific article

A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors

scientific article published on 29 June 2010

AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa

scientific article published in August 2003

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.

scientific article published on December 2011

Absence of γ-chain in keratinocytes alters chemokine secretion resulting in reduced immune cell recruitment

scientific article

Actin cytoskeletal defects in immunodeficiency

scientific article

Activating WASP mutations associated with X-linked neutropenia result in enhanced actin polymerization, altered cytoskeletal responses, and genomic instability in lymphocytes

scientific article

Activating mutations of N-WASP alter Shigella pathogenesis

scientific article published on 18 April 2009

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

scientific article published on 19 October 2018

Altered BCR and TLR signals promote enhanced positive selection of autoreactive transitional B cells in Wiskott-Aldrich syndrome

scientific article

Autoimmune lymphoproliferative syndrome: molecular basis of disease and clinical phenotype

scientific article

Autoinflammatory periodic fever, immunodeficiency, and thrombocytopenia (PFIT) caused by mutation in actin-regulatory gene WDR1

scientific article

Automated manufacturing of chimeric antigen receptor T cells for adoptive immunotherapy using CliniMACS prodigy

scientific article published on August 2016

Autonomous role of Wiskott-Aldrich Syndrome platelet deficiency in inducing autoimmunity and inflammation

scientific article published on 5 February 2018

Averting abnormal inheritance: potential of gene therapy and preimplantation diagnosis

scientific article published on January 2004

B cell-intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice.

scientific article published in February 2012

B-cell intrinsic TLR7 signals promote depletion of the marginal zone in a murine model of Wiskott-Aldrich syndrome

scientific article

Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection

scientific article published on 23 December 2016

Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells

scientific article

Bleeding and splenectomy in Wiskott-Aldrich syndrome: A single-centre experience

scientific article published on 23 July 2018

Bone marrow-derived IFN-producing killer dendritic cells account for the tumoricidal activity of unpulsed dendritic cells

scientific article published on November 2008

Bone morphogenetic protein 4 modulates c-Kit expression and differentiation potential in murine embryonic aorta-gonad-mesonephros haematopoiesis in vitro

scientific article published on October 2007

Bone morphogenetic protein-4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA

article

Brief report: self-organizing neuroepithelium from human pluripotent stem cells facilitates derivation of photoreceptors.

scientific article published in February 2013

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

Catechins and antitumor immunity: Not MDSC's cup of tea.

scientific article published on 08 May 2013

Cellular control of cortical actin nucleation

scientific article

Characterization of a core region in the A2UCOE that confers effective anti-silencing activity

scientific article published on 31 August 2017

Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency

scientific article

Codon optimization of human factor VIII cDNAs leads to high-level expression

scientific article published in November 2010

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

scientific article published on 01 November 2018

Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies

scientific article published on 21 July 2008

Coherence analysis discriminates between retroviral integration patterns in CD34(+) cells transduced under differing clinical trial conditions

scientific article published on 29 April 2015

Common variable immunodeficiency and natural killer cell lymphopenia caused by Ets-binding site mutation in the IL-2 receptor γ (IL2RG) gene promoter

scientific article published on 30 October 2015

Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells

scientific article published on September 2005

Comprehensive genomic access to vector integration in clinical gene therapy

scientific article published on 22 November 2009

Concise review: MicroRNAs as modulators of stem cells and angiogenesis

scientific article published on May 2014

Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases

scientific article

Construction of stable packaging cell lines for clinical lentiviral vector production

scientific article published on 12 March 2015

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

scientific article

Correction of both immunodeficiency and hypoparathyroidism by thymus transplantation in complete DiGeorge syndrome

scientific article published on 30 October 2019

Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene

scientific article

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

scientific article

Corrigendum to "Local Administration of an Adeno-Associated Viral Vector Expressing IL-10 Reduces Monocyte Infiltration and Subsequent Photoreceptor Damage During Experimental Autoimmune Uveitis".

scientific article

Corrigendum to "Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice".

scientific article published on 3 November 2005

Current and emerging treatment options for Wiskott-Aldrich syndrome

scientific article

Cytoskeletal remodeling mediated by WASp in dendritic cells is necessary for normal immune synapse formation and T-cell priming

scientific article published on 20 June 2011

Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment

scientific article

Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells

scientific article published on 18 July 2016

Development of anti-PAX3 immune responses; a target for cancer immunotherapy

scientific article published on 23 February 2007

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

scientific article published on 22 December 2009

Development of lentiviral gene therapy for Wiskott Aldrich syndrome

Diminished production of anti-inflammatory mediators during neutrophil apoptosis and macrophage phagocytosis in chronic granulomatous disease (CGD).

scientific article published in May 2003

Disease-associated missense mutations in the EVH1 domain disrupt intrinsic WASp function causing dysregulated actin dynamics and impaired dendritic cell migration

scientific article

EROS mutations: decreased NADPH oxidase function and chronic granulomatous disease

scientific article published on 09 October 2018

Editorial overview: New technologies

scientific article published on 28 September 2015

Effect of gene therapy on visual function in Leber's congenital amaurosis

scientific article

Effective gene therapy with nonintegrating lentiviral vectors

scientific article

Emerging Neuronal Precursors from Amniotic Fluid–Derived Down Syndrome Induced Pluripotent Stem Cells

scientific article published on 01 August 2014

Emotional and behavioural difficulties in chronic granulomatous disease

Engraftment defect of cytokine-cultured adult human mobilized CD34(+) cells is related to reduced adhesion to bone marrow niche elements

scientific article

Enhancement of integrin-mediated transfection of haematopoietic cells with a synthetic vector system

scientific article

Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy

scientific article published on 29 August 2017

Essential role of the NADPH oxidase subunit p47(phox) in endothelial cell superoxide production in response to phorbol ester and tumor necrosis factor-alpha

scientific article published on February 2002

Evolving Gene Therapy in Primary Immunodeficiency

scientific article

Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts

scientific article

Exacerbated experimental arthritis in Wiskott-Aldrich syndrome protein deficiency: modulatory role of regulatory B cells

scientific article

Excess F-actin mechanically impedes mitosis leading to cytokinesis failure in X-linked neutropenia by exceeding Aurora B kinase error correction capacity

scientific article published on 12 September 2012

FOXP3+ Tregs require WASP to restrain Th2-mediated food allergy

scientific article

Failure of SCID-X1 gene therapy in older patients

scientific article

Fetal haemopoietic cells display enhanced migration across endothelium

scientific article published on 01 February 2002

Functional gap junctions accumulate at the immunological synapse and contribute to T cell activation

scientific article

Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells

scientific article published on 18 May 2011

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

scientific article published in August 2007

Gene correction of induced pluripotent stem cells derived from a murine model of X-linked chronic granulomatous disorder

scientific article published on January 2014

Gene therapy for Fanconi anemia: one step closer to the clinic.

scientific article published on February 2012

Gene therapy for PIDs: progress, pitfalls and prospects

scientific article

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

scientific article published on 17 July 2017

Gene therapy for inherited immunodeficiencies.

scientific article

Gene therapy for lympho-hematopoietic disorders.

scientific article

Gene therapy for monogenic disorders of the bone marrow

scientific article published on 05 June 2015

Gene therapy for ocular angiogenesis.

scientific article

Gene therapy for primary immunodeficiencies

scientific article

Gene therapy for primary immunodeficiencies

scientific article published on May 2008

Gene therapy for primary immunodeficiency

scientific article published on December 2011

Gene therapy for severe combined immune deficiency

scientific article published on 2 July 2004

Gene therapy for severe combined immunodeficiencies

scientific article

Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency

scientific article published on February 2012

Gene therapy for the Wiskott-Aldrich syndrome

scientific article

Gene therapy in primary immunodeficiencies

scientific article published in July 2005

Gene therapy matures in the clinic

scientific article

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

scientific article

Gene therapy of chronic granulomatous disease: the engraftment dilemma

scientific article

Gene therapy of inherited immunodeficiencies

article

Gene therapy: X-SCID transgene leukaemogenicity

scientific article

Gene therapy: great expectations?

scientific article

Gene therapy: is IL2RG oncogenic in T-cell development?

scientific article published in September 2006

Gene therapy: progress and predictions

scientific article

Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells

scientific article

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

scientific article

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

scientific article published on 4 October 2017

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

scientific article published in August 2011

Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein.

scientific article published on February 2008

High plasticity of pediatric adipose tissue-derived stem cells: too much for selective skeletogenic differentiation?

scientific article

High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter

scientific article published in May 2002

High-titer stocks of adeno-associated virus from replicating amplicons and herpes vectors.

scientific article

Hot spots of retroviral integration in human CD34+ hematopoietic cells

scientific article

Human Amniocytes Are Receptive to Chemically Induced Reprogramming to Pluripotency

scientific article

Human gamma delta T cells: a lymphoid lineage cell capable of professional phagocytosis

scientific article

Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression

scientific article published on January 2012

Human mid-trimester amniotic fluid stem cells cultured under embryonic stem cell conditions with valproic acid acquire pluripotent characteristics

scientific article published on 27 November 2012

Hybrid lentiviral vectors

scientific article published on 20 April 2010

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

scientific article

Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ.

scientific article published on 27 March 2014

Immunodeficiency and severe susceptibility to bacterial infection associated with a loss-of-function homozygous mutation of MKL1.

scientific article

Impact of BREXIT on UK Gene and Cell Therapy: The Need for Continued Pan-European Collaboration.

scientific article

Impaired T-cell priming in vivo resulting from dysfunction of WASp-deficient dendritic cells

scientific article published on 02 August 2007

Impaired bone marrow homing of cytokine-activated CD34+ cells in the NOD/SCID model

scientific article published on 20 November 2003

Impaired dendritic-cell homing in vivo in the absence of Wiskott-Aldrich syndrome protein

scientific article published on 19 October 2004

Impaired neutrophil migration and phagocytosis in IRAK-4 deficiency

scientific article

Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy

scientific article

Inhibition of calpain stabilises podosomes and impairs dendritic cell motility

scientific article (publication date: June 2006)

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 mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

scientific article published on September 2008

Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

scientific article

Integrase-deficient lentiviral vectors mediate efficient gene transfer to human vascular smooth muscle cells with minimal genotoxic risk

scientific article

Integrin linked kinase (ILK) regulates podosome maturation and stability in dendritic cells.

scientific article published on 06 February 2014

LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia

scientific article published on 14 September 2012

Lentivectors are efficient tools to manipulate the dendritic cell cytoskeleton

scientific article published on 29 July 2011

Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB.

scientific article published on January 2016

Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I.

scientific article published on 08 April 2016

Lentiviral gene therapy against human immunodeficiency virus type 1, using a novel human TRIM21-cyclophilin A restriction factor.

scientific article

Lentiviral gene therapy for X-linked chronic granulomatous disease

scientific article published on 27 January 2020

Lentiviral vector integration profiles differ in rodent postmitotic tissues

scientific article

Lentiviral vector transduction of spermatozoa as a tool for the study of early development

scientific article

Lentiviral vectors can be used for full-length dystrophin gene therapy

scientific article

Lentiviral vectors can be used for full-length dystrophin gene therapy.

scientific article published on 17 March 2017

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

scientific article published on 24 April 2007

Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction

scientific article published in February 2007

Lentivirus technologies for modulation of the immune system

scientific article published on 09 September 2015

Lentivirus-mediated reprogramming of somatic cells in the absence of transgenic transcription factors

scientific article

Leukocyte adhesion deficiency-I: A comprehensive review of all published cases

scientific article published on 20 January 2018

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

scientific article published on 10 December 2016

Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis

scientific article

Long-Term Hematopoietic Engraftment of Congenic Amniotic Fluid Stem Cells After in Utero Intraperitoneal Transplantation to Immune Competent Mice.

scientific article

Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy

scientific article published in September 2003

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

scientific article published on 12 March 2021

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 persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency

scientific article published in August 2011

Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy

scientific article published on October 2002

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

scientific article published on 24 January 2022

Loss of the interleukin-6 receptor causes immunodeficiency, atopy, and abnormal inflammatory responses

scientific article published on 24 June 2019

Loss-of-function nuclear factor κB subunit 1 (NFKB1) variants are the most common monogenic cause of common variable immunodeficiency in Europeans ⬇️

scientific article published on 22 February 2018

Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration

scientific article

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

scientific article published on 30 March 2010

Maturation of DC is associated with changes in motile characteristics and adherence

scientific article published on 01 February 2004

Mechanisms of WASp-mediated hematologic and immunologic disease.

scientific article published on 12 August 2004

Megakaryocytes assemble podosomes that degrade matrix and protrude through basement membrane

scientific article published on 10 January 2013

Modelling of human Wiskott-Aldrich syndrome protein mutants in zebrafish larvae using in vivo live imaging

scientific article

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

scientific article published in January 2017

Moving forward on shifting sands: ethical regulation of gene therapy clinical trials in the United kingdom

scientific article published on April 2013

Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells

scientific article published in April 2007

Myogenic cell proliferation and generation of a reversible tumorigenic phenotype are triggered by preirradiation of the recipient site

scientific article

N-WASP is required for B-cell-mediated autoimmunity in Wiskott-Aldrich syndrome.

scientific article

New insights into the biology of Wiskott-Aldrich syndrome (WAS).

scientific article

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.

scientific article published on 17 April 2018

Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy

scientific article published on 28 January 2009

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

scientific article

One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years

scientific article published on 25 July 2018

Out of harm's way

scientific article published on 01 January 2011

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

scientific article published on April 2015

Patching up hematopoietic stem cells

scientific article published on 01 December 2015

Patients with T+/lowNK+IL-2 receptor γ chain deficiency have differentially-impaired cytokine signaling resulting in severe combined immunodeficiency

scientific article published on 28 August 2014

Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency

scientific article published on 19 December 2014

Phase I study protocol for ex-vivo lentiviral gene therapy for the inherited skin disease, Netherton Syndrome

scientific article published on 18 October 2013

Phosphorylation of WASp is a key regulator of activity and stability in vivo

scientific article published on September 2009

Platelet actin nodules are podosome-like structures dependent on Wiskott-Aldrich syndrome protein and ARP2/3 complex.

scientific article

Polyphenon [corrected] E enhances the antitumor immune response in neuroblastoma by inactivating myeloid suppressor cells

scientific article published on 15 January 2013

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

scientific article

Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency

scientific article

Prevalence and clinical challenges among adults with primary immunodeficiency and recombination-activating gene deficiency. ⬇️

scientific article published on 22 February 2018

Primary Immune Deficiency Treatment Consortium (PIDTC) report

scientific article published on 15 October 2013

Primary immunodeficiencies due to abnormalities of the actin cytoskeleton

scientific article

Progress and Prospects: Advancements in Retroviral Vector Design, Generation, and Application

scientific article published on 01 October 2011

Progress and prospects for engineered T cell therapies

scientific article

Progress in Lentiviral Vector Technologies

scientific article published on 01 February 2013

Publisher Correction: Whole-genome sequencing of a sporadic primary immunodeficiency cohort

scientific article published on 17 July 2020

RISC control for gene therapy

scientific article published in June 2006

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

scientific article

Recent advances in the understanding of genetic defects of neutrophil number and function

scientific article published on 31 August 2010

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

Reduced type I interferon production by dendritic cells and weakened antiviral immunity in patients with Wiskott-Aldrich syndrome protein deficiency

scientific article

Relevance of biallelic versus monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes

scientific article

Repeated courses of rituximab for autoimmune cytopenias may precipitate profound hypogammaglobulinaemia requiring replacement intravenous immunoglobulin

scientific article published on 9 May 2009

Restoring the natural tropism of AAV2 vectors for human liver

scientific article published on 01 September 2020

Retrovirus and lentivirus vector design and methods of cell conditioning

scientific article published in January 2012

Risks of gene therapy should be weighed against lack of alternatives for many diseases

scientific article published on April 2005

Role of WASP in cell polarity and podosome dynamics of myeloid cells

scientific article published on 06 July 2010

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

scientific article published on 05 December 2012

SAP mediates specific cytotoxic T-cell functions in X-linked lymphoproliferative disease

scientific article published on 15 January 2004

Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing

scientific article

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

scientific article published on 8 January 2008

Sequence Analysis of TNFRSF13b, Encoding TACI, in Patients with Systemic Lupus Erythematosus

scientific article published on 27 April 2007

Severe cutaneous papillomavirus disease after haematopoietic stem-cell transplantation in patients with severe combined immunodeficiency

scientific article published on 01 October 2004

Sheep CD34+ amniotic fluid cells have hematopoietic potential and engraft after autologous in utero transplantation

scientific article published in January 2015

Signal transducer and activator of transcription 5 tyrosine phosphorylation for the diagnosis and monitoring of patients with severe combined immunodeficiency

scientific article published in February 2009

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

scientific article

Sleeping beauty transposition from nonintegrating lentivirus

scientific article

Stable gene transfer to muscle using non-integrating lentiviral vectors

scientific article published on 14 August 2007

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

scientific article

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning

scientific article

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

scientific article (publication date: 18 April 2002)

T cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents Haemophagocytic Lymphohistiocytosis manifestations.

scientific article published on 17 January 2018

T-cell reconstitution after thymus xenotransplantation induces hair depigmentation and loss

scientific article

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

scientific article published on 12 August 2020

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model

scientific article published on 29 September 2017

The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice

scientific article published on 31 March 2011

The Wiskott-Aldrich syndrome

scientific article published on 01 April 2006

The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function.

scientific article published on 23 March 2009

The Wiskott-Aldrich syndrome: The actin cytoskeleton and immune cell function

scientific article

The cytoplasm of living cells behaves as a poroelastic material

scientific article published on 6 January 2013

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis

scientific article published on 13 November 2012

The functional role of S/MARs in episomal vectors as defined by the stress-induced destabilization profile of the vector sequences

scientific article published on 25 February 2009

The future of gene therapy

scientific article

The impact of retroviral suicide gene transduction procedures on T cells

scientific article published in November 2003

The leukocyte podosome

scientific article

The tight junction associated signalling proteins ZO-1 and ZONAB regulate retinal pigment epithelium homeostasis in mice

scientific article

The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity

scientific article

Thymus transplantation for complete DiGeorge syndrome: European experience.

scientific article

Tolerance induction using lentiviral gene delivery delays onset and severity of collagen II arthritis

scientific article

Towards gene therapy for EBV-associated posttransplant lymphoma with genetically modified EBV-specific cytotoxic T cells

scientific article

Transatlantic consortium spotlights need for changes in gene therapy trials

scientific article published on November 2010

Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease.

scientific article published on 27 April 2018

Treating Immunodeficiency through HSC Gene Therapy

scientific 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

Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia

scientific article published on 27 June 2006

Tyrosine phosphorylation of WASP promotes calpain-mediated podosome disassembly

scientific article published on December 2011

Tyrosine phosphorylation of WIP releases bound WASP and impairs podosome assembly in macrophages

scientific article

Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia

scientific article published on 27 August 2007

Update on clinical gene therapy in childhood

scientific article published on November 2007

Update on the hyper immunoglobulin M syndromes

scientific article (publication date: 23 February 2010)

Use of compassionate-case ATMP in preclinical data for clinical trial applications

scientific article published in June 2012

Use of nonintegrating lentiviral vectors for gene therapy

scientific article published on June 2007

Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach

scientific article

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

scientific article published in August 2007

WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis.

scientific article

WASP: a key immunological multitasker

scientific article

WASp deficiency in mice results in failure to form osteoclast sealing zones and defects in bone resorption

scientific article published on 15 January 2004

WASp in immune-system organization and function

scientific article

WASp-dependent actin cytoskeleton stability at the dendritic cell immunological synapse is required for extensive, functional T cell contacts.

scientific article

WIP regulates the stability and localization of WASP to podosomes in migrating dendritic cells

scientific article published on December 2006

Whole-genome sequencing of a sporadic primary immunodeficiency cohort

scientific article published on 06 May 2020

Wiskott Aldrich syndrome protein regulates non-selective autophagy and mitochondrial homeostasis in human myeloid cells

scientific article published on 02 November 2020

Wiskott-Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation

scientific article published on 22 July 2009

Wiskott-Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells

scientific article

Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis

scientific article published on 07 August 2008

Wiskott-Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response

article

Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells

scientific article

Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor

scientific article published on 26 February 2013

Wiskott-Aldrich syndrome protein: Emerging mechanisms in immunity

scientific article published on 14 August 2017

X-linked lymphoproliferative disease: clinical, diagnostic and molecular perspective

scientific article

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

scientific article

iPSCs: Unstable origins?

scientific article published on July 2011

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