Current and emerging treatment options for Wiskott-Aldrich syndrome (Q33424297)

26 July 2017

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title

Current and emerging treatment options for Wiskott-Aldrich syndrome (English)

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26 July 2017

main subject

Wiskott-Aldrich syndrome

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Adrian J Thrasher

2

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Austen Worth

series ordinal

1

object named as

Austen J J Worth

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26 July 2017

publication date

9 July 2015

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Expert Review of Clinical Immunology

26 July 2017

published in

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26 July 2017

volume

11

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26 July 2017

issue

9

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26 July 2017

page(s)

1015-1032

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Isolation of a novel gene mutated in Wiskott-Aldrich syndrome

26 July 2017

cites work

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Autoinhibition and activation mechanisms of the Wiskott-Aldrich syndrome protein

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WIP regulates N-WASP-mediated actin polymerization and filopodium formation

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

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WIP is a chaperone for Wiskott-Aldrich syndrome protein (WASP).

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Mechanism of recruitment of WASP to the immunological synapse and of its activation following TCR ligation

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Wiskott-Aldrich syndrome protein is a key regulator of the phagocytic cup formation in macrophages

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Direct observation of dendritic actin filament networks nucleated by Arp2/3 complex and WASP/Scar proteins.

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Fyn and PTP-PEST-mediated regulation of Wiskott-Aldrich syndrome protein (WASp) tyrosine phosphorylation is required for coupling T cell antigen receptor engagement to WASp effector function and T cell activation

21 January 2018

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Phosphorylation of tyrosine 291 enhances the ability of WASp to stimulate actin polymerization and filopodium formation. Wiskott-Aldrich Syndrome protein

21 January 2018

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Phosphorylation of the WASP-VCA domain increases its affinity for the Arp2/3 complex and enhances actin polymerization by WASP

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Contingent phosphorylation/dephosphorylation provides a mechanism of molecular memory in WASP.

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

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Ubiquitylation-dependent negative regulation of WASp is essential for actin cytoskeleton dynamics

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T-cell receptor ligation causes Wiskott-Aldrich syndrome protein degradation and F-actin assembly downregulation

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N-WASP deficiency reveals distinct pathways for cell surface projections and microbial actin-based motility

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Wiskott Aldrich syndrome protein (WASP) and N-WASP are critical for T cell development.

21 January 2018

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Nuclear role of WASp in gene transcription is uncoupled from its ARP2/3-dependent cytoplasmic role in actin polymerization.

21 January 2018

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The open conformation of WASP regulates its nuclear localization and gene transcription in myeloid cells

21 January 2018

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Nuclear role of WASp in the pathogenesis of dysregulated TH1 immunity in human Wiskott-Aldrich syndrome

21 January 2018

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Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells

21 January 2018

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WASP regulates suppressor activity of human and murine CD4(+)CD25(+)FOXP3(+) natural regulatory T cells

21 January 2018

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Phenotypic perturbation of B cells in the Wiskott-Aldrich syndrome.

21 January 2018

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

21 January 2018

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WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function

21 January 2018

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Molecular and phenotypic abnormalities of B lymphocytes in patients with Wiskott-Aldrich syndrome

21 January 2018

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Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans

21 January 2018

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WASp-deficient B cells play a critical, cell-intrinsic role in triggering autoimmunity

21 January 2018

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Autoimmunity in Wiskott-Aldrich syndrome

21 January 2018

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A multiinstitutional survey of the Wiskott-Aldrich syndrome.

21 January 2018

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X-linked thrombocytopenia in three males with normal sized platelets due to novel WAS gene mutations

21 January 2018

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Clinical course of patients with WASP gene mutations

21 January 2018

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Genotype-proteotype linkage in the Wiskott-Aldrich syndrome

21 January 2018

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

21 January 2018

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Autoimmune manifestations of the wiskott-aldrich syndrome

21 January 2018

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Development of IgA nephropathy-like glomerulonephritis associated with Wiskott–Aldrich syndrome protein deficiency

21 January 2018

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The thrombocytopenia of WAS: a familial form of ITP?

21 January 2018

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Flow cytometric analysis of platelets from childrenwith the Wiskott‐Aldrich syndrome reveals defects in platelet development, activation and structure

21 January 2018

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Occurrence of Aortic Aneurysms in 5 Cases of Wiskott-Aldrich Syndrome

21 January 2018

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Immunodeficiency-associated lymphomas

21 January 2018

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Wiskott-Aldrich syndrome presenting with a clinical picture mimicking juvenile myelomonocytic leukaemia

21 January 2018

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Wiskott-Aldrich syndrome with unusual clinical features similar to juvenile myelomonocytic leukemia

21 January 2018

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Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment

21 January 2018

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Constitutively activating mutation in WASP causes X-linked severe congenital neutropenia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Flow cytometric determination of intracytoplasmic Wiskott-Aldrich syndrome protein in peripheral blood lymphocyte subpopulations

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Absence of expression of the Wiskott-Aldrich syndrome protein in peripheral blood cells of Wiskott-Aldrich syndrome patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

WASP (Wiskott-Aldrich syndrome protein) gene mutations and phenotype

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Mutations of the Wiskott-Aldrich Syndrome Protein (WASP): hotspots, effect on transcription, and translation and phenotype/genotype correlation

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

A novel primary human immunodeficiency due to deficiency in the WASP-interacting protein WIP

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

X-linked thrombocytopenia caused by a mutation in the Wiskott-Aldrich syndrome (WAS) gene that disrupts interaction with the WAS protein (WASP)-interacting protein (WIP)

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Structure of the N-WASP EVH1 Domain-WIP Complex

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Identification of WASP mutations, mutation hotspots and genotype-phenotype disparities in 24 patients with the Wiskott-Aldrich syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Monozygotic Twin Pair Showing Discordant Phenotype for X-linked Thrombocytopenia and Wiskott–Aldrich Syndrome: a Role for Epigenetics?

21 January 2018

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https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Combined de-novo mutation and non-random X-chromosome inactivation causing Wiskott-Aldrich syndrome in a female with thrombocytopenia.

21 January 2018

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https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Wiskott-Aldrich syndrome in a female

21 January 2018

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https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Somatic mosaicism in the Wiskott-Aldrich syndrome: molecular and functional characterization of genotypic revertants

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Evolution of highly polymorphic T cell populations in siblings with the Wiskott-Aldrich Syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis of function and distribution in lymphoid organs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Mosaicism of NK cells in a patient with Wiskott-Aldrich syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

The spleen in the Wiskott-Aldrich syndrome: histopathologic abnormalities of the white pulp correlate with the clinical phenotype of the disease.

21 January 2018

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An international study examining therapeutic options used in treatment of Wiskott-Aldrich syndrome

21 January 2018

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Intravenous immunoglobulin, splenectomy, and antibiotic prophylaxis in Wiskott-Aldrich syndrome

21 January 2018

1 reference

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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

21 January 2018

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https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Complete Correction of the Wiskott-Aldrich Syndrome by Allogeneic Bone-Marrow Transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors

21 January 2018

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Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.

21 January 2018

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

21 January 2018

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Outcomes of hematopoietic stem cell transplantation in primary immunodeficiency: a report from the Australian and New Zealand Children's Haematology Oncology Group and the Australasian Bone Marrow Transplant Recipient Registry

21 January 2018

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21 January 2018

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HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders

21 January 2018

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Cellular and molecular basis of haploidentical hematopoietic stem cell transplantation in the successful treatment of high-risk leukemias: role of alloreactive NK cells

21 January 2018

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Reduced-intensity conditioning for allogeneic stem cell transplant in primary immune deficiencies

21 January 2018

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Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

21 January 2018

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Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression

21 January 2018

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Outcomes of donor lymphocyte infusion for treatment of mixed donor chimerism after a reduced-intensity preparative regimen for pediatric patients with nonmalignant diseases

21 January 2018

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Impaired in vitro regulatory T cell function associated with Wiskott-Aldrich syndrome

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Wiskott-Aldrich syndrome protein is required for regulatory T cell homeostasis

21 January 2018

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The Wiskott-Aldrich syndrome protein is required for the function of CD4(+)CD25(+)Foxp3(+) regulatory T cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

Wiskott-Aldrich syndrome protein is required for homeostasis and function of invariant NKT cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F1744666X.2015.1062366

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

21 January 2018

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Malignancies after Hematopoietic Cell Transplantation for Primary Immune Deficiencies: A Report from the Center for International Blood and Marrow Transplant Research

21 January 2018

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Pre-emptive rituximab based on viraemia and T cell reconstitution: a highly effective strategy for the prevention of Epstein-Barr virus-associated lymphoproliferative disease following stem cell transplantation

21 January 2018

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Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.

21 January 2018

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Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity

21 January 2018

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Efficacy of gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

21 January 2018

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

21 January 2018

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

21 January 2018

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Disruption of hSWI/SNF complexes in T cells by WAS mutations distinguishes X-linked thrombocytopenia from Wiskott-Aldrich syndrome

21 January 2018

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A peptide derived from the Wiskott-Aldrich syndrome (WAS) protein-interacting protein (WIP) restores WAS protein level and actin cytoskeleton reorganization in lymphocytes from patients with WAS mutations that disrupt WIP binding

21 January 2018

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Successful haematopoietic stem cell transplantation in 44 children from healthy siblings conceived after preimplantation HLA matching

21 January 2018

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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

21 January 2018

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

21 January 2018

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NMR Analyses of the Activation of the Arp2/3 Complex by Neuronal Wiskott−Aldrich Syndrome Protein†

21 January 2018

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Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience

21 January 2018

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10.1586/1744666X.2015.1062366

21 January 2018

Identifiers

DOI

1 reference

26 July 2017

1 reference