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Engineering Protein-Secreting Plasma Cells by Homology-Directed Repair in Primary Human B Cells

Molecular therapy, 2018-02, Vol.26 (2), p.456-467

2018

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Antigen‐specific in vitro expansion of factor VIII‐specific regulatory T cells induces tolerance in hemophilia A mice

Journal of thrombosis and haemostasis, 2020-02, Vol.18 (2), p.328-340

2020

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Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

Molecular therapy. Nucleic acids, 2020-06, Vol.20, p.534-544

2020

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Comparative glycosylation mapping of plasma-derived and recombinant human factor VIII

PloS one, 2020-05, Vol.15 (5), p.e0233576-e0233576

2020

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Effect of chimeric antigen receptor T cells against protease-activated receptor 1 for treating pancreatic cancer

BMC medicine, 2023-09, Vol.21 (1), p.338-19, Article 338

2023

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Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Blood, 2014-08, Vol.124 (6), p.913-923

2014

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Development of therapeutic microbubbles for enhancing ultrasound-mediated gene delivery

Journal of controlled release, 2014-05, Vol.182, p.111-120

2014

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Microbubbles and ultrasound increase intraventricular polyplex gene transfer to the brain

Journal of controlled release, 2016-06, Vol.231, p.86-93

2016

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A Treg-Selective IL-2 Mutein Prevents the Formation of Factor VIII Inhibitors in Hemophilia Mice Treated With Factor VIII Gene Therapy

Frontiers in immunology, 2020-04, Vol.11, p.638-638

2020

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Engineering Chimeric Antigen Receptor T Cells against Immune Checkpoint Inhibitors PD-1/PD-L1 for Treating Pancreatic Cancer

Molecular therapy. Oncolytics, 2020-06, Vol.17, p.571-585

2020

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Ex Vivo Expanded Human Vγ9Vδ2 T-Cells Can Suppress Epithelial Ovarian Cancer Cell Growth

International journal of molecular sciences, 2019-03, Vol.20 (5), p.1139

2019

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Induction of long-term tolerance to a specific antigen using anti-CD3 lipid nanoparticles following gene therapy

Molecular therapy. Nucleic acids, 2023-12, Vol.34, p.102043, Article 102043

2023

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Ultrasound-mediated gene delivery of factor VIII plasmids for hemophilia A gene therapy in mice

Molecular therapy. Nucleic acids, 2022-03, Vol.27, p.916-926

2022

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Strategies to target long-lived plasma cells for treating hemophilia A inhibitors

Cellular immunology, 2016-03, Vol.301, p.65-73

2016

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Site-Specific N- and O-Glycosylation Analysis of Human Plasma Fibronectin

Frontiers in chemistry, 2021-06, Vol.9, p.691217-691217

2021

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Long-Term and Therapeutic-Level Hepatic Gene Expression of Human Factor IX after Naked Plasmid Transfer in Vivo

Molecular therapy, 2001-06, Vol.3 (6), p.947-957

2001

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Ultrasound-Mediated Gene Therapy in Swine Livers Using Single-Element, Multi-lensed, High-Intensity Ultrasound Transducers

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.179-188

2018

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Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

Nature genetics, 1997-07, Vol.16 (3), p.270-276

1997

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Ultrasound enhances gene delivery of human factor IX plasmid

Human gene therapy, 2005-07, Vol.16 (7), p.893-905

2005

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Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors

Thrombosis journal, 2016-10, Vol.14 (Suppl 1), p.41-41, Article 41

Miao, Carol H

2016

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Prolonging pulse duration in ultrasound-mediated gene delivery lowers acoustic pressure threshold for efficient gene transfer to cells and small animals

Journal of controlled release, 2018-06, Vol.279, p.345-354

2018

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Progress toward inducing immunologic tolerance to factor VIII

Blood, 2013-05, Vol.121 (22), p.4449-4456

2013

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CD4+ T cells engineered with FVIII-CAR and murine Foxp3 suppress anti-factor VIII immune responses in hemophilia a mice

Cellular immunology, 2020-12, Vol.358, p.104216-104216, Article 104216

2020

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Blunting specific T-dependent antibody responses with engineered “decoy” B cells

Molecular therapy, 2024-08

2024

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Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A

Molecular therapy, 2015-04, Vol.23 (4), p.617-626

2015

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Engineering Protein-Secreting Plasma Cells by Homology-Directed Repair in Primary Human B Cells

Antigen‐specific in vitro expansion of factor VIII‐specific regulatory T cells induces tolerance in hemophilia A mice

Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

Comparative glycosylation mapping of plasma-derived and recombinant human factor VIII

Effect of chimeric antigen receptor T cells against protease-activated receptor 1 for treating pancreatic cancer

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Development of therapeutic microbubbles for enhancing ultrasound-mediated gene delivery

Microbubbles and ultrasound increase intraventricular polyplex gene transfer to the brain

A Treg-Selective IL-2 Mutein Prevents the Formation of Factor VIII Inhibitors in Hemophilia Mice Treated With Factor VIII Gene Therapy

Engineering Chimeric Antigen Receptor T Cells against Immune Checkpoint Inhibitors PD-1/PD-L1 for Treating Pancreatic Cancer

Ex Vivo Expanded Human Vγ9Vδ2 T-Cells Can Suppress Epithelial Ovarian Cancer Cell Growth

Induction of long-term tolerance to a specific antigen using anti-CD3 lipid nanoparticles following gene therapy

Ultrasound-mediated gene delivery of factor VIII plasmids for hemophilia A gene therapy in mice

Strategies to target long-lived plasma cells for treating hemophilia A inhibitors

Site-Specific N- and O-Glycosylation Analysis of Human Plasma Fibronectin

Long-Term and Therapeutic-Level Hepatic Gene Expression of Human Factor IX after Naked Plasmid Transfer in Vivo

Ultrasound-Mediated Gene Therapy in Swine Livers Using Single-Element, Multi-lensed, High-Intensity Ultrasound Transducers

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

Ultrasound enhances gene delivery of human factor IX plasmid

Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors

Prolonging pulse duration in ultrasound-mediated gene delivery lowers acoustic pressure threshold for efficient gene transfer to cells and small animals

Progress toward inducing immunologic tolerance to factor VIII

CD4+ T cells engineered with FVIII-CAR and murine Foxp3 suppress anti-factor VIII immune responses in hemophilia a mice

Blunting specific T-dependent antibody responses with engineered “decoy” B cells

Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A

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