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Platelet-Targeted Gene Therapy for Hemophilia

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.100-108

Shi, Qizhen

2018

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A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII

Blood, 2014-06, Vol.123 (24), p.3706-3713

2014

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Platelet-Targeted FVIII Gene Therapy Restores Hemostasis and Induces Immune Tolerance for Hemophilia A

Frontiers in immunology, 2020-06, Vol.11, p.964-964

2020

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Hemophilia a patients with inhibitors: Mechanistic insights and novel therapeutic implications

Frontiers in immunology, 2022-12, Vol.13, p.1019275

2022

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In vivo generated hematopoietic stem cells from genome edited induced pluripotent stem cells are functional in platelet-targeted gene therapy of murine hemophilia A

Haematologica (Roma), 2020-04, Vol.105 (4), p.e175-e179

2020

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Association of Platelet Desialylation and Circulating Follicular Helper T Cells in Patients With Thrombocytopenia

Frontiers in immunology, 2022-04, Vol.13, p.810620-810620

2022

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Targeting transmembrane-domain-less MOG expression to platelets prevents disease development in experimental autoimmune encephalomyelitis

Frontiers in immunology, 2022-10, Vol.13, p.1029356-1029356

2022

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Utilizing Platelets as a Targeted for Gene Therapy of Hemophilia a and Hemophilia B

Microscopy and microanalysis, 2020-08, Vol.26 (S2), p.826-827

Shi, Qizhen

2020

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Platelet gene therapy induces robust immune tolerance even in a primed model via peripheral clonal deletion of antigen-specific T cells

Molecular therapy. Nucleic acids, 2021-03, Vol.23, p.719-730

2021

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Preparation and characterization of BaTiO3 powders and ceramics by sol-gel process using oleic acid as surfactant

Materials science & engineering. A, Structural materials : properties, microstructure and processing, 2008-01, Vol.473 (1-2), p.34-41

2008

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Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice

Blood advances, 2019-09, Vol.3 (18), p.2700-2711

2019

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Desialylated Platelet Clearance in the Liver is a Novel Mechanism of Systemic Immunosuppression

Research (Washington), 2023, Vol.6, p.0236-0236

2023

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Platelet Gene Therapy Promotes Targeted Peripheral Tolerance by Clonal Deletion and Induction of Antigen-Specific Regulatory T Cells

Frontiers in immunology, 2018-09, Vol.9, p.1950-1950

2018

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Factor IX ectopically expressed in platelets can be stored in α-granules and corrects the phenotype of hemophilia B mice

Blood, 2010-08, Vol.116 (8), p.1235-1243

2010

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Induction of activated T follicular helper cells is critical for anti-FVIII inhibitor development in hemophilia A mice

Blood advances, 2019-10, Vol.3 (20), p.3099-3110

2019

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From Hydrogen-Bonded Net-to-Net Framework to Twofold Interpenetrated (4,6) Net: Effect of Ligand Topology on the Supramolecular Structural Diversity

Crystal growth & design, 2007-02, Vol.7 (2), p.300-306

2007

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Correction of Murine Bernard–Soulier Syndrome by Lentivirus-mediated Gene Therapy

Molecular therapy, 2012-03, Vol.20 (3), p.625-632

2012

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The severe spontaneous bleeding phenotype in a novel hemophilia A rat model is rescued by platelet FVIII expression

Blood advances, 2020-01, Vol.4 (1), p.55-65

2020

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The impact of von Willebrand factor on factor VIII memory immune responses

Blood advances, 2017-08, Vol.1 (19), p.1565-1574

2017

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Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond

Journal of thrombosis and haemostasis, 2024-01, Vol.22 (1), p.23-34

2024

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Formation of Two-Dimensional Supramolecular Water Layer Containing (H2O)18 Morphology via Dianion Templating

Crystal growth & design, 2006-04, Vol.6 (4), p.812-814

2006

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Platelet αIIbβ3 Receptor Antagonist Impedes the Hemostatic Efficacy of Platelet-Specific FVIII Gene Therapy in Hemophilia a Mice

Blood, 2022-11, Vol.140 (Supplement 1), p.2712-2712

2022

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Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment

Blood, 2003-12, Vol.102 (12), p.4006-4013

2003

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A rat model of severe VWD by elimination of the VWF gene using CRISPR/Cas9

Research and practice in thrombosis and haemostasis, 2020-01, Vol.4 (1), p.64-71

2020

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TGF-β1 along with other platelet contents augments Treg cells to suppress anti-FVIII immune responses in hemophilia A mice

Blood advances, 2016-12, Vol.1 (2), p.139-151

2016

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Platelet-Targeted Gene Therapy for Hemophilia

A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII

Platelet-Targeted FVIII Gene Therapy Restores Hemostasis and Induces Immune Tolerance for Hemophilia A

Hemophilia a patients with inhibitors: Mechanistic insights and novel therapeutic implications

In vivo generated hematopoietic stem cells from genome edited induced pluripotent stem cells are functional in platelet-targeted gene therapy of murine hemophilia A

Association of Platelet Desialylation and Circulating Follicular Helper T Cells in Patients With Thrombocytopenia

Targeting transmembrane-domain-less MOG expression to platelets prevents disease development in experimental autoimmune encephalomyelitis

Utilizing Platelets as a Targeted for Gene Therapy of Hemophilia a and Hemophilia B

Platelet gene therapy induces robust immune tolerance even in a primed model via peripheral clonal deletion of antigen-specific T cells

Preparation and characterization of BaTiO3 powders and ceramics by sol-gel process using oleic acid as surfactant

Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice

Desialylated Platelet Clearance in the Liver is a Novel Mechanism of Systemic Immunosuppression

Platelet Gene Therapy Promotes Targeted Peripheral Tolerance by Clonal Deletion and Induction of Antigen-Specific Regulatory T Cells

Factor IX ectopically expressed in platelets can be stored in α-granules and corrects the phenotype of hemophilia B mice

Induction of activated T follicular helper cells is critical for anti-FVIII inhibitor development in hemophilia A mice

From Hydrogen-Bonded Net-to-Net Framework to Twofold Interpenetrated (4,6) Net: Effect of Ligand Topology on the Supramolecular Structural Diversity

Correction of Murine Bernard–Soulier Syndrome by Lentivirus-mediated Gene Therapy

The severe spontaneous bleeding phenotype in a novel hemophilia A rat model is rescued by platelet FVIII expression

The impact of von Willebrand factor on factor VIII memory immune responses

Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond

Formation of Two-Dimensional Supramolecular Water Layer Containing (H2O)18 Morphology via Dianion Templating

Platelet αIIbβ3 Receptor Antagonist Impedes the Hemostatic Efficacy of Platelet-Specific FVIII Gene Therapy in Hemophilia a Mice

Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment

A rat model of severe VWD by elimination of the VWF gene using CRISPR/Cas9

TGF-β1 along with other platelet contents augments Treg cells to suppress anti-FVIII immune responses in hemophilia A mice

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