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Enhancing the pharmaceutical properties of protein drugs by ancestral sequence reconstruction

Nature biotechnology, 2017-01, Vol.35 (1), p.35-37

2017

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Platelet clearance via shear-induced unfolding of a membrane mechanoreceptor

Nature communications, 2016-09, Vol.7 (1), p.12863-12863, Article 12863

2016

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Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A

Human gene therapy, 2018-10, Vol.29 (10), p.1183-1201

2018

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Development of chimeric antigen receptors targeting T-cell malignancies using two structurally different anti-CD5 antigen binding domains in NK and CRISPR-edited T cell lines

Oncoimmunology, 2018-03, Vol.7 (3), p.e1407898-e1407898

2018

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Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity

Nature communications, 2023-07, Vol.14 (1), p.4206-4206, Article 4206

2023

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Dissecting the cellular components of ex vivo γδ T cell expansions to optimize selection of potent cell therapy donors for neuroblastoma immunotherapy trials

Oncoimmunology, 2022-12, Vol.11 (1), p.2057012-2057012

2022

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The Immune Response to the fVIII Gene Therapy in Preclinical Models

Frontiers in immunology, 2020-04, Vol.11, p.494

2020

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Directing the migration of serum-free, ex vivo -expanded Vγ9Vδ2 T cells

Frontiers in immunology, 2024-02, Vol.15, p.1331322-1331322

2024

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Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII

Frontiers in immunology, 2022-12, Vol.13, p.954984

2022

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Ligand-based targeting of c-kit using engineered γδ T cells as a strategy for treating acute myeloid leukemia

Frontiers in immunology, 2023, Vol.14, p.1294555-1294555

2023

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Structure of Blood Coagulation Factor VIII in Complex With an Anti-C2 Domain Non-Classical, Pathogenic Antibody Inhibitor

Frontiers in immunology, 2021-06, Vol.12, p.697602-697602

2021

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Ex vivo expanded patient-derived γδ T-cell immunotherapy enhances neuroblastoma tumor regression in a murine model

Oncoimmunology, 2019, Vol.8 (8), p.1593804-1593804

2019

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Neutralizing Antibodies Against Factor VIII Can Occur Through a Non-Germinal Center Pathway

Frontiers in immunology, 2022-05, Vol.13, p.880829-880829

2022

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Enhancing the effectiveness of γδ T cells by mRNA transfection of chimeric antigen receptors or bispecific T cell engagers

Molecular therapy. Oncolytics, 2023-06, Vol.29, p.145-157

2023

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High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Blood advances, 2019-10, Vol.3 (19), p.2883-2894

2019

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Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.57-69

2018

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Potentiation of thrombin generation in hemophilia A plasma by coagulation factor VIII and characterization of antibody-specific inhibition

PloS one, 2012-10, Vol.7 (10), p.e48172

2012

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Retroviral modification of mesenchymal stem cells for gene therapy of hemophilia

Methods in molecular biology (Clifton, N.J.), 2008, Vol.433, p.203

Doering, Christopher B

2008

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Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.98-107

2021

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Development and characterization of recombinant ovine coagulation factor VIII

PloS one, 2012-11, Vol.7 (11), p.e49481

2012

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Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII

Molecular therapy. Methods & clinical development, 2021-06, Vol.21, p.710-727

2021

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Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A

Frontiers in cell and developmental biology, 2021-08, Vol.9, p.678117-678117

2021

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Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.465-477

2020

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Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

Molecular therapy, 2009-07, Vol.17 (7), p.1145-1154

2009

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Characterization of Donor Variability for γδ T Cell ex vivo Expansion and Development of an Allogeneic γδ T Cell Immunotherapy

Frontiers in medicine, 2020-11, Vol.7, p.588453-588453

2020

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Enhancing the pharmaceutical properties of protein drugs by ancestral sequence reconstruction

Platelet clearance via shear-induced unfolding of a membrane mechanoreceptor

Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A

Development of chimeric antigen receptors targeting T-cell malignancies using two structurally different anti-CD5 antigen binding domains in NK and CRISPR-edited T cell lines

Transplanting FVIII/ET3-secreting cells in fetal sheep increases FVIII levels long-term without inducing immunity or toxicity

Dissecting the cellular components of ex vivo γδ T cell expansions to optimize selection of potent cell therapy donors for neuroblastoma immunotherapy trials

The Immune Response to the fVIII Gene Therapy in Preclinical Models

Directing the migration of serum-free, ex vivo -expanded Vγ9Vδ2 T cells

Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII

Ligand-based targeting of c-kit using engineered γδ T cells as a strategy for treating acute myeloid leukemia

Structure of Blood Coagulation Factor VIII in Complex With an Anti-C2 Domain Non-Classical, Pathogenic Antibody Inhibitor

Ex vivo expanded patient-derived γδ T-cell immunotherapy enhances neuroblastoma tumor regression in a murine model

Neutralizing Antibodies Against Factor VIII Can Occur Through a Non-Germinal Center Pathway

Enhancing the effectiveness of γδ T cells by mRNA transfection of chimeric antigen receptors or bispecific T cell engagers

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A

Potentiation of thrombin generation in hemophilia A plasma by coagulation factor VIII and characterization of antibody-specific inhibition

Retroviral modification of mesenchymal stem cells for gene therapy of hemophilia

Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

Development and characterization of recombinant ovine coagulation factor VIII

Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII

Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A

Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A

Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

Characterization of Donor Variability for γδ T Cell ex vivo Expansion and Development of an Allogeneic γδ T Cell Immunotherapy

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