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Journal Articles

Factor VIII Expression from a Novel F8 Transgene through a Lentiviral Vector Transduced CD34+ Autologous Hematopoietic Stem Cells for Gene Therapy of Severe Hemophilia Α: Final Results from a Phase 1 Clinical Trial

Blood (2024) 144 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2024-202882

Published: 2024

Journal Articles

F5-Atlanta induced Exitron Splicing

Karen L Zimowski, Maya Maarouf, Julie R Wechsler, Carlos Lozano, Seema R Patel, Shannon L Meeks, Christopher B. Doering

Blood (2024) 144 (Supplement 1): 429.

https://doi.org/10.1182/blood-2024-208391

Published: 2024

Journal Articles

Cryoem Structures and Conformational Landscapes of Activated Blood Coagulation Factor VIII and the Intrinsic Tenase Complex

Kenneth C Childers, Omar Davulcu, Carmen Helena Coxon, Pete Lollar, Christopher B. Doering, P. Clint Spiegel, Jr.

Blood (2024) 144 (Supplement 1): 428.

https://doi.org/10.1182/blood-2024-211229

Published: 2024

Journal Articles

Ancestral L-Asparaginases Are Cytotoxic Against ALL Cell Lines with Predicted Reduced Immunogenicity

Kristopher Adrian Knight, Harrison C. Brown, Mathew S. Karpen, H. Trent Spencer, Christopher B. Doering, Sunil S Raikar

Blood (2024) 144 (Supplement 1): 4169.

https://doi.org/10.1182/blood-2024-205342

Published: 2024

Journal Articles

Cryo-EM Structure of Coagulation Factor VIII Bound to a Patient-Derived Anti-A2 Domain Antibody Inhibitor

Kenneth C Childers, Jordan Vaughan, John S. Lollar, Christopher B Doering, Carmen Helena Coxon, P. Clint Spiegel, Jr.

Blood (2023) 142 (Supplement 1): 2598.

https://doi.org/10.1182/blood-2023-182304

Published: 2023

Journal Articles

Assessment of Transduction of CD34+ Human Hematopoietic Stem Cells from Patients with Severe Hemophilia-Α with Lentiviral Vector Carrying a High Expression FVIII Transgene (CD68-ET3-LV)

Gurbind Singh, Mohanashankar A M, Shaji R Velayudhan, Gabriela Denning, Abraham Sunder Singh, Anbu Jeba, Aby Abraham, Christopher B Doering, H Trent Spencer, Alok Srivastava

Blood (2023) 142 (Supplement 1): 481.

https://doi.org/10.1182/blood-2023-188513

Published: 2023

Journal Articles

Development of a Novel L-Asparaginase with Enhanced Properties Using Ancestral Sequence Reconstruction

Kristopher A Knight, Harrison C. Brown, Kinnede R. White, H. Trent Spencer, Christopher B Doering, Sunil S. Raikar

Blood (2023) 142 (Supplement 1): 4191.

https://doi.org/10.1182/blood-2023-190281

Published: 2023

Journal Articles

Structure of coagulation factor VIII bound to a patient-derived anti–C1 domain antibody inhibitor

Kenneth C. Childers, Nathan G. Avery, Kevin A. Estrada Alamo, Omar Davulcu, Rose Marie Haynes, Pete Lollar, Christopher B. Doering, Carmen H. Coxon, P. Clint Spiegel, Jr.

Blood (2023) 142 (2): 197–201.

https://doi.org/10.1182/blood.2023020181

Published: 2023

Includes: Supplemental data

Journal Articles

Nonhuman glycans can regulate anti–factor VIII antibody formation in mice

Connie M. Arthur, Patricia E. Zerra, Sooncheon Shin, Jianmei Wang, Xeuzheng Song, Christopher B. Doering, Pete Lollar, Shannon Meeks, Sean R. Stowell

Blood (2022) 139 (9): 1312–1317.

https://doi.org/10.1182/blood.2020009210

Published: 2022

Includes: Supplemental data

Journal Articles

In Utero Transplantation of FVIII-Expressing Human Placental Cells Upregulates Gene Pathways Associated with Immune Tolerance, Altering the Response to Postnatal FVIII Infusion

Martin Rodriguez, Brady Trevisan, Sunil George, Jordan E Shields, Jorge Figueroa, Diane Meares, John Owen, Anthony Atala, Christopher B Doering, H. Trent Spencer, Christopher D Porada, M. Graca Almeida-Porada

Blood (2021) 138 (Supplement 1): 1863.

https://doi.org/10.1182/blood-2021-153417

Published: 2021

Journal Articles

Administration of FVIII-Expressing Human Placental Cells to Juvenile Sheep Yields Multi-Organ Engraftment, Therapeutic Plasma FVIII Levels and Alter Immune Signaling Pathways to Evade FVIII Inhibitor Induction

Brady Trevisan, Martin Rodriguez, Jacqueline Dizon, Sunil George, Jordan E Shields, Shannon Lankford, Rebecca Combs, John Owen, Anthony Atala, Christopher B Doering, H. Trent Spencer, Christopher D Porada, M. Graca Almeida-Porada

Blood (2021) 138 (Supplement 1): 3966.

https://doi.org/10.1182/blood-2021-151207

Published: 2021

Journal Articles

SAXS Analysis of the Intrinsic Tenase Complex Bound to Lipid Nanodisc Highlights Intermolecular Contacts between Factors VIIIa/IXa

Kenneth C Childers, Shaun Peters, John S. Lollar, H. Trent Spencer, Christopher B Doering, Paul Clinton Spiegel

Blood (2021) 138 (Supplement 1): 2099.

https://doi.org/10.1182/blood-2021-147073

Published: 2021

Journal Articles

Structure of blood coagulation factor VIII in complex with an anti–C1 domain pathogenic antibody inhibitor

Joseph S. Gish, Lexi Jarvis, Kenneth C. Childers, Shaun C. Peters, Connor S. Garrels, Ian W. Smith, H. Trent Spencer, Christopher B. Doering, Pete Lollar, P. Clint Spiegel, Jr

Blood (2021) 137 (21): 2981–2986.

https://doi.org/10.1182/blood.2020008940

Published: 2021

Includes: Supplemental data

Journal Articles

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia a in a Large Animal Model Demonstrates Absence of Maternal Exposure to the Cells or Gene Products Infused into the Fetus

Quan DM Pham, Martin Rodriguez, BSc, Ritu M Ramamurthy, Sunil George, PhD, Jorge Figueroa, MD, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 32.

https://doi.org/10.1182/blood-2020-140883

Published: 2020

Journal Articles

Administration of Cells Engineered to Secrete Fviii-mcoET3 in Prenatal Sheep Recipients Results in Sustained Curative Fviii Plasma Levels for 3 Years after Birth, without Immune or Toxicity-Related Adverse Events

Martin Rodriguez, BSc, Brady Trevisan, Sunil George, PhD, Ritu M Ramamurthy, Andrew Rabah, Jordan E Shields, MS, Denise Schwahn, DVM, Jorge Figueroa, MD, Diane Meares, John Owen, MDMBA,FRCPC, Michael Gautreaux, PhD, D.ABHI, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 1–2.

https://doi.org/10.1182/blood-2020-140845

Published: 2020

Journal Articles

Delivery of Fviii-mcoET3 to Previously Untreated Sheep Using Human Placental Cells Enables Durable Elevation of Plasma FVIII Levels and Avoids Inhibitor Formation

Brady Trevisan, Martin Rodriguez, BSc, Sunil George, PhD, Shannon Lankford, Jordan E Shields, MS, Jorge Figueroa, MD, Diane Meares, John Owen, MDMBA,FRCPC, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD, M. Graca Almeida-Porada, MD PhD

Blood (2020) 136 (Supplement 1): 34.

https://doi.org/10.1182/blood-2020-140745

Published: 2020

Journal Articles

The 3.2 Angstrom Crystal Structure of a Bioengineered Factor VIII Construct Illustrates Two Conformations of the C2 Domain

Ian W Smith, MS, Anne d'Aquino, BS, Ernest T. Parker, Christopher B Doering, PhD, Paul C Spiegel, Jr., PhD

Blood (2018) 132 (Supplement 1): 21.

https://doi.org/10.1182/blood-2018-99-119083

Published: 2018

Journal Articles

Engineering CD5-Targeted Chimeric Antigen Receptors and Edited T Cells for the Treatment of T-Cell Leukemia

Lauren C Fleischer, Sunil S Raikar, MD, Robert Moot, B.S., Kristopher A Knight, B.S., Christopher B Doering, PhD, H. Trent Spencer, PhD

Blood (2017) 130 (Supplement 1): 1914.

https://doi.org/10.1182/blood.V130.Suppl_1.1914.1914

Published: 2017

Journal Articles

Prenatal Transplantation of Placental Cells Engineered to Express FVIII Leads to Corrective Plasma Levels of FVIII after Birth

M. Graca Almeida-Porada, MD PhD, Sunil George, PhD, Alshaima Morsi, MD, Nadia El-Akabawy, MD, Jorge Figueroa, Jordan E Shields, MS, Diane Meares, Andrew M. Farland, MD, Anthony Atala, MD, Christopher B Doering, PhD, H. Trent Spencer, PhD, Christopher D Porada, PhD

Blood (2017) 130 (Supplement 1): 361.

https://doi.org/10.1182/blood.V130.Suppl_1.361.361

Published: 2017

Journal Articles

Factor V Atlanta: A Novel Mutation in the F5 Gene Reveals Potential New Cis-Acting Elements Involved in Regulating FV-Short and TFPI Levels

Karen L Zimowski, MD, Michelle D. Ho, BS, Jordan E Shields, MS, Gabriela Denning, PhD, Teodolinda Petrillo, PhD, Navdeep Jhita, MD, Angel A Rivera, PhD, Miguel A Escobar, MD, Christine L Kempton, Christopher B Doering, PhD, Rodney M Camire, PhD

Blood (2017) 130 (Supplement 1): 366.

https://doi.org/10.1182/blood.V130.Suppl_1.366.366

Published: 2017

NARROW

Factor VIII Expression from a Novel F8 Transgene through a Lentiviral Vector Transduced CD34+ Autologous Hematopoietic Stem Cells for Gene Therapy of Severe Hemophilia Α: Final Results from a Phase 1 Clinical Trial

F5-Atlanta induced Exitron Splicing

Cryoem Structures and Conformational Landscapes of Activated Blood Coagulation Factor VIII and the Intrinsic Tenase Complex

Ancestral L-Asparaginases Are Cytotoxic Against ALL Cell Lines with Predicted Reduced Immunogenicity

Cryo-EM Structure of Coagulation Factor VIII Bound to a Patient-Derived Anti-A2 Domain Antibody Inhibitor

Assessment of Transduction of CD34+ Human Hematopoietic Stem Cells from Patients with Severe Hemophilia-Α with Lentiviral Vector Carrying a High Expression FVIII Transgene (CD68-ET3-LV)

Development of a Novel L-Asparaginase with Enhanced Properties Using Ancestral Sequence Reconstruction

Structure of coagulation factor VIII bound to a patient-derived anti–C1 domain antibody inhibitor

Nonhuman glycans can regulate anti–factor VIII antibody formation in mice

In Utero Transplantation of FVIII-Expressing Human Placental Cells Upregulates Gene Pathways Associated with Immune Tolerance, Altering the Response to Postnatal FVIII Infusion

Administration of FVIII-Expressing Human Placental Cells to Juvenile Sheep Yields Multi-Organ Engraftment, Therapeutic Plasma FVIII Levels and Alter Immune Signaling Pathways to Evade FVIII Inhibitor Induction

SAXS Analysis of the Intrinsic Tenase Complex Bound to Lipid Nanodisc Highlights Intermolecular Contacts between Factors VIIIa/IXa

Structure of blood coagulation factor VIII in complex with an anti–C1 domain pathogenic antibody inhibitor

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia a in a Large Animal Model Demonstrates Absence of Maternal Exposure to the Cells or Gene Products Infused into the Fetus

Administration of Cells Engineered to Secrete Fviii-mcoET3 in Prenatal Sheep Recipients Results in Sustained Curative Fviii Plasma Levels for 3 Years after Birth, without Immune or Toxicity-Related Adverse Events

Delivery of Fviii-mcoET3 to Previously Untreated Sheep Using Human Placental Cells Enables Durable Elevation of Plasma FVIII Levels and Avoids Inhibitor Formation

The 3.2 Angstrom Crystal Structure of a Bioengineered Factor VIII Construct Illustrates Two Conformations of the C2 Domain

Engineering CD5-Targeted Chimeric Antigen Receptors and Edited T Cells for the Treatment of T-Cell Leukemia

Prenatal Transplantation of Placental Cells Engineered to Express FVIII Leads to Corrective Plasma Levels of FVIII after Birth

Factor V Atlanta: A Novel Mutation in the F5 Gene Reveals Potential New Cis-Acting Elements Involved in Regulating FV-Short and TFPI Levels

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American Society of Hematology

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American Society of Hematology

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