Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. - sprookjes - yvandenbroek - TriplyDB

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

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

http://www.wikidata.org/entity/Q37089728

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The contribution of mouse models to the understanding of constitutional thrombocytopenia Advances of gene therapy for primary immunodeficiencies Inherited platelet disorders: toward DNA-based diagnosis Mechanism-Based Strategies for the Management of Autoimmunity and Immune Dysregulation in Primary Immunodeficiencies Retroviral DNA Integration Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?Current and emerging treatment options for Wiskott-Aldrich syndrome.A rare case of Wiskott-Aldrich Syndrome with normal platelet size: a case report A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome Eltrombopag use in a patient with Wiskott-Aldrich syndrome.Use of Lentiviral Particles As a Cell Membrane-Based mFasL Delivery System for In Vivo Treatment of Inflammatory Arthritis.Gene Therapy 2017: Progress and Future Directions.INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human Cells Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates.Systemic epigenetic response to recombinant lentiviral vectors independent of proviral integration In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects.Neural Stem Cells Engineered to Express Three Therapeutic Factors Mediate Recovery from Chronic Stage CNS Autoimmunity Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis.Gene therapy for primary immune deficiencies: a Canadian perspective.Building the foundation for genomics in precision medicine.Gene therapy for monogenic disorders of the bone marrow.Reconstructing the immune system with lentiviral vectors.Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector High Level of Perforin Expression Is Required for Effective Correction of Hemophagocytic Lymphohistiocytosis.Reliable RT-qPCR-based titration of retroviral and lentiviral vectors via quantification of residual vector plasmid DNA in samples.Gene Therapy for the Treatment of Primary Immune Deficiencies.Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world.INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions.Modern management of phagocyte defects.Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View.Advances in clinical immunology in 2015.New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing.Evolving Gene Therapy in Primary Immunodeficiency.Gene Therapy for β-Hemoglobinopathies.Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry.Primary immunodeficiency diseases: Genomic approaches delineate heterogeneous Mendelian disorders.A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene Therapies.

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

http://www.wikidata.org/entity/Q37089728

description

article científic

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article scientifique

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articolo scientifico

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artigo científico

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bilimsel makale

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scientific article published on April 2015

@en

vedecký článok

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vetenskaplig artikel

@sv

videnskabelig artikel

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vědecký článek

@cs

name

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

@en

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

@nl

type

label

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

@en

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

@nl

prefLabel

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

@en

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

@nl

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Journal of the American Medical Association

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

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Anne Galy

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Anne-Marie McNicol

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Annick Lim

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Charles Berry

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Christine Rivat

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Eric A Sherman

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Frances Male

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Francois Lefrère

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Frederic D Bushman

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Géraldine Honnet

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P2860

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

Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation

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

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

Efficacy of gene therapy for X-linked severe combined immunodeficiency

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

Selection of target sites for mobile DNA integration in the human genome

HIV-1 integration in the human genome favors active genes and local hotspots

Actin cytoskeletal function is spared, but apoptosis is increased, in WAS patient hematopoietic cells.

Clinical course of patients with WASP gene mutations.

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1550-1563

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scholarly article

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10.1001/JAMA.2015.3253

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15

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313

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Marina Cavazzana-Calvo

Capucine Picard

Salima Hacein-Bey-Abina

Emmanuelle M. Six

Laure Caccavelli

Jean-Marc Tréluyer

Sébastien Héritier

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2015-04-01T00:00:00Z

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25898053

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