Efficacy of gene therapy for X-linked severe combined immunodeficiency - sprookjes - yvandenbroek - TriplyDB

Efficacy of gene therapy for X-linked severe combined immunodeficiency

Efficacy of gene therapy for X-linked severe combined immunodeficiency

http://www.wikidata.org/entity/Q28288838

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The therapeutic potential of genome editing for β-thalassemia Adoptive T-Cell Immunotherapy State-of-the-art human gene therapy: part I. Gene delivery technologies Use of V(D)J recombination excision circles to identify T- and B-cell defects and to monitor the treatment in primary and acquired immunodeficiencies Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models Gene therapy for primary immunodeficiencies Retroviral integrations in gene therapy trials Adipocytes as a vehicle for ex vivo gene therapy: Novel replacement therapy for diabetes and other metabolic diseases Gene therapy studies in a canine model of X-linked severe combined immunodeficiency Genetics of SCID Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives Human RAG mutations: biochemistry and clinical implications Gene Therapy for Metabolic Diseases A novel intranuclear RNA vector system for long-term stem cell modification Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012 Smart micro/nanoparticles in stimulus-responsive drug/gene delivery systems.Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts Proteomic profiling of salivary gland after nonviral gene transfer mediated by conventional plasmids and minicircles.Preparation and testing of quaternized chitosan nanoparticles as gene delivery vehicles.Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.Gene therapy for PIDs: progress, pitfalls and prospects Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors.Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.Current and emerging treatment options for Wiskott-Aldrich syndrome.Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases.Dynamics of HSPC repopulation in nonhuman primates revealed by a decade-long clonal-tracking study.Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites.SiRNA Delivery with PEGylated Graphene Oxide Nanosheets for Combined Photothermal and Genetherapy for Pancreatic Cancer.Comparing DNA integration site clusters with scan statistics.Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy.Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases.Curing genetic disease with gene therapy.Serial number tagging reveals a prominent sequence preference of retrotransposon integration MicroRNA 142-3p attenuates spread of replicating retroviral vector in hematopoietic lineage-derived cells while maintaining an antiviral immune response.T-cell Receptor and K-deleting Recombination Excision Circles in Newborn Screening of T- and B-cell Defects: Review of the Literature and Future Challenges.Estimating abundances of retroviral insertion sites from DNA fragment length data.

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Efficacy of gene therapy for X-linked severe combined immunodeficiency

http://www.wikidata.org/entity/Q28288838

description

2010 nî lūn-bûn

@nan

2010 թուականի Յուլիսին հրատարակուած գիտական յօդուած

@hyw

2010 թվականի հուլիսին հրատարակված գիտական հոդված

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2010年の論文

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2010年論文

@yue

2010年論文

@zh-hant

2010年論文

@zh-hk

2010年論文

@zh-mo

2010年論文

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2010年论文

@wuu

name

Efficacy of gene therapy for X-linked severe combined immunodeficiency

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Efficacy of gene therapy for X-linked severe combined immunodeficiency

@en

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@nl

type

label

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@ast

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@en

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@nl

prefLabel

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@ast

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@en

Efficacy of gene therapy for X-linked severe combined immunodeficiency

@nl

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The New England Journal of Medicine

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Efficacy of gene therapy for X-linked severe combined immunodeficiency

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P2093

Annick Lim

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Bernd H Belohradsky

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Chantal Martinache

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Charles C Berry

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Gary P Wang

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Jean Laurent Casanova

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Julia Hauer

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Lily Leiva

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Ricardo Sorensen

http://www.w3.org/2001/XMLSchema#string

Stephane Blanche

http://www.w3.org/2001/XMLSchema#string

P2860

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

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

Human interleukin 10 induces naive surface immunoglobulin D+ (sIgD+) B cells to secrete IgG1 and IgG3

The interleukin-2 receptor gamma chain: its role in the multiple cytokine receptor complexes and T cell development in XSCID

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

B cell-intrinsic signaling through IL-21 receptor and STAT3 is required for establishing long-lived antibody responses in humans.

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

Cytokines and immunodeficiency diseases.

Interleukin 13 induces interleukin 4-independent IgG4 and IgE synthesis and CD23 expression by human B cells.

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

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355-64

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scholarly article

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1368887

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10.1056/NEJMOA1000164

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P433

4

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363

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Marina Cavazzana-Calvo

Capucine Picard

Salima Hacein-Bey-Abina

Frederic D. Bushman

Frédéric Rieux-Laucat

Marianne Debré

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2010-07-22T00:00:00Z

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20660403

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2957288

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