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

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

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

http://www.wikidata.org/entity/Q34356904

about

Exploiting Genetic Interference for Antiviral Therapy Advances of gene therapy for primary immunodeficiencies Clinical development of gene therapy: results and lessons from recent successes Human induced pluripotent stem cells for monogenic disease modelling and therapy Production of lentiviral vectors Current and future alternative therapies for beta-thalassemia major Recent advances in treatment of severe primary immunodeficiencies Targeted approaches to induce immune tolerance for Pompe disease therapy Megakaryocyte- and megakaryocyte precursor-related gene therapies Gene therapy: progress and predictions Cell-based therapy technology classifications and translational challenges Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation Bone Marrow Gene Therapy for HIV/AIDS Perinatal systemic gene delivery using adeno-associated viral vectors Synthetic biology in mammalian cells: next generation research tools and therapeutics Primary cilia in the developing and mature brain Advances in understanding and treating dystrophic epidermolysis bullosa Gene therapy on the move Unraveling the repertoire in wiskott-Aldrich syndrome The diagnostic approach to monogenic very early onset inflammatory bowel disease Advances in unrelated and alternative donor hematopoietic cell transplantation for nonmalignant disorders Systemic delivery of microRNA-101 potently inhibits hepatocellular carcinoma in vivo by repressing multiple targets Genome editing: the road of CRISPR/Cas9 from bench to clinic Genetically modified mesenchymal stromal cells in cancer therapy Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors Advantages and applications of CAR-expressing natural killer cells Manufacture of tumor- and virus-specific T lymphocytes for adoptive cell therapies Therapeutic genome editing: prospects and challenges Genetic engineering of hematopoiesis for targeted IFN-α delivery inhibits breast cancer progression Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo Retroviral DNA Integration A novel intranuclear RNA vector system for long-term stem cell modification Charting a clear path: the ASGCT Standardized Pathways Conference DNA transposon-based gene vehicles - scenes from an evolutionary drive Development of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical Practice Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?Spatiotemporal control of gene expression in bone-marrow derived cells of the tumor microenvironment induced by MRI guided focused ultrasound.Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts Enhancers are major targets for murine leukemia virus vector integration

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

http://www.wikidata.org/entity/Q34356904

description

2013 nî lūn-bûn

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2013 թուականի Յուլիսին հրատարակուած գիտական յօդուած

@hyw

2013 թվականի հուլիսին հրատարակված գիտական հոդված

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2013年の論文

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2013年論文

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2013年論文

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2013年論文

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2013年論文

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2013年論文

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2013年论文

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name

Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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type

label

Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

@nl

prefLabel

Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

@ast

Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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SCIENCE.1233151

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Lentiviral hematopoietic stem ...... with Wiskott-Aldrich syndrome.

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Alessandra Biffi

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Andrea Assanelli

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Anne Galy

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Ayse Metin

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Claudia Benati

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Clelia Di Serio

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Costanza Evangelio

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Cristina Baricordi

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Danilo Pellin

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David J Dow

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P2860

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Retroviral integrations in gene therapy trials

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

Efficacy of gene therapy for X-linked severe combined immunodeficiency

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

Hot spots of retroviral integration in human CD34+ hematopoietic cells

Wiskott-Aldrich syndrome protein (WASP) and N-WASP are critical for peripheral B-cell development and function

GREAT improves functional interpretation of cis-regulatory regions

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

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

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1233151

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10.1126/SCIENCE.1233151

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341

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Eugenio Montini

Andrea Calabria

Alessandro Aiuti

Maria Grazia Roncarolo

Francesca Ferrua

Maria Carmina Castiello

Andrea Finocchi

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2013-07-11T00:00:00Z

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248705010

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