Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. - Test2 - elhamkhani - TriplyDB

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

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

http://www.wikidata.org/entity/Q33413888

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A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells Advances of gene therapy for primary immunodeficiencies Clinical development of gene therapy: results and lessons from recent successes In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application Recent advances in treatment of severe primary immunodeficiencies Progress and prospects for engineered T cell therapies Unraveling the repertoire in wiskott-Aldrich syndrome PSMA-specific CAR-engineered T cells eradicate disseminated prostate cancer in preclinical models Advantages and applications of CAR-expressing natural killer cells Retroviral DNA Integration Charting a clear path: the ASGCT Standardized Pathways Conference Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?Diagnosis and treatment of inherited thrombocytopenias.Assessment of Immature Platelet Fraction in the Diagnosis of Wiskott-Aldrich Syndrome.Current and emerging treatment options for Wiskott-Aldrich syndrome.Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.Vector integration and tumorigenesis.Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy.Succession of transiently active tumor-initiating cell clones in human pancreatic cancer xenografts.Gene Therapy 2017: Progress and Future Directions.Platelets: still a therapeutical target for haemostatic disorders.Adoptive T-cell therapy: adverse events and safety switches.Quantitative shearing linear amplification polymerase chain reaction: an improved method for quantifying lentiviral vector insertion sites in transplanted hematopoietic cell systems Cell cycle status of CD34(+) hemopoietic stem cells determines lentiviral integration in actively transcribed and development-related genes.Clinical applications of gene therapy for primary immunodeficiencies.What has happened in the last 50 years in immunology.adLIMS: a customized open source software that allows bridging clinical and basic molecular research studies Genetic barcode sequencing for screening altered population dynamics of hematopoietic stem cells transduced with lentivirus Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy.Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4(+) T Cells.Gamma-Retrovirus Integration Marks Cell Type-Specific Cancer Genes: A Novel Profiling Tool in Cancer Genomics Gene activity in primary T cells infected with HIV89.6: intron retention and induction of genomic repeats.Towards a Safer, More Randomized Lentiviral Vector Integration Profile Exploring Artificial LEDGF Chimeras.INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis.Multilineage polyclonal engraftment of Cal-1 gene-modified cells and in vivo selection after SHIV infection in a nonhuman primate model of AIDS Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes.

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P2860

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

http://www.wikidata.org/entity/Q33413888

description

2014 nî lūn-bûn

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2014 թուականի Մարտին հրատարակուած գիտական յօդուած

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2014 թվականի մարտին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

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

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

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

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

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prefLabel

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

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

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SCITRANSLMED.3007280

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Science Translational Medicine

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

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P2093

Adrian Schwarzer

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Anna Paruzynski

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Chris Fraser

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Christoph Klein

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Doris Steinemann

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Gudrun Göhring

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Hanno Glimm

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Irina Kondratenko

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Kaan Boztug

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Klaus Kühlcke

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P2860

Isolation of a novel gene mutated in Wiskott-Aldrich syndrome

A census of human cancer genes

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

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

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

Efficacy of gene therapy for X-linked severe combined immunodeficiency

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

Measurement of Diversity

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

Wiskott-Aldrich syndrome protein is required for NK cell cytotoxicity and colocalizes with actin to NK cell-activating immunologic synapses.

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227ra33

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scholarly article

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10.1126/SCITRANSLMED.3007280

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227

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6

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Christof von Kalle

Christian J Braun

Brigitte Schlegelberger

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2014-03-01T00:00:00Z

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260759632

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24622513

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Wiskott-Aldrich syndrome