Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A. - Wikidata - awgldk - TriplyDB

Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A.

Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A.

http://www.wikidata.org/entity/Q33713758

about

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application Hemophilia A: an ideal disease to correct in utero Gene therapy for hemophilia Engineered drug resistant γδ T cells kill glioblastoma cell lines during a chemotherapy challenge: a strategy for combining chemo- and immunotherapy Development and characterization of recombinant ovine coagulation factor VIII Engineered drug-resistant immunocompetent cells enhance tumor cell killing during a chemotherapy challenge.Clinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A.In vivo efficacy of platelet-delivered, high specific activity factor VIII variants Advancements in gene transfer-based therapy for hemophilia A.Simplified prototyping of perfusable polystyrene microfluidics.In vitro and In vivo Model Systems for Hemophilia A Gene Therapy.Lentiviral vector platform for production of bioengineered recombinant coagulation factor VIII Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitors Enhanced biosynthesis of coagulation factor VIII through diminished engagement of the unfolded protein response.Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery.Role of bone marrow transplantation for correcting hemophilia A in mice.Treatment of a solid tumor using engineered drug-resistant immunocompetent cells and cytotoxic chemotherapy.Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.Engineering Factor Viii for Hemophilia Gene Therapy.Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models.Ectopic platelet-delivered factor (F) VIII for the treatment of Hemophilia A: Plasma and platelet FVIII, is it all the same?Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A.Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity.Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells.Pathophysiology, diagnosis and prevention of arthropathy in patients with haemophilia.Platelet and endothelial expression of clotting factors for the treatment of hemophilia.Potential for cellular stress response to hepatic factor VIII expression from AAV vector Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A.Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A.

P2860

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P2860

Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A.

http://www.wikidata.org/entity/Q33713758

description

2009 nî lūn-bûn

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2009 թուականի Մարտին հրատարակուած գիտական յօդուած

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2009 թվականի մարտին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年论文

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Directed engineering of a high ...... gene therapy of hemophilia A.

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Directed engineering of a high ...... gene therapy of hemophilia A.

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Directed engineering of a high ...... gene therapy of hemophilia A.

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Molecular Therapy

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Directed engineering of a high ...... gene therapy of hemophilia A.

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P2093

Bagirath Gangadharan

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Christopher B Doering

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David A McCarty

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Gabriela Denning

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H Trent Spencer

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Jennifer M Johnston

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Keith W Kerstann

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Kerry Dooriss

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P2860

Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots

Antigenicity of putative phospholipid membrane-binding residues in factor VIII.

Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A.

Preclinical gene therapy studies for hemophilia using adenoviral vectors.

Onco-retroviral and lentiviral vector-based gene therapy for hemophilia: preclinical studies.

Clinical gene transfer studies for hemophilia A.

Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer.

Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens.

Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A

Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein.

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1145-1154

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10.1038/MT.2009.35

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7

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17

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24175206

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19259064

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