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Journal Articles

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Giang N. Nguyen, BS, Lauren E. Wimsey, DVM, Elizabeth P. Merricks, PhD, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 3628.

https://doi.org/10.1182/blood-2019-126085

Published: 2019

Journal Articles

Cryo-EM Structure of BIVV001 Reveals Coagulation Factor VIII-Von Willebrand Factor D'D3 Interaction Mode

James Fuller, PhD, Joseph Batchelor, PhD, Kevin Knockenhauer, PhD, Hans-Peter Biemann, PhD, Robert Peters

Blood (2019) 134 (Supplement_1): 94.

https://doi.org/10.1182/blood-2019-126645

Published: 2019

Journal Articles

Neutralizing and Non-Neutralizing Anti-FVIII Antibodies in Black and White Hemophilia A Subjects: A Natural History Profile

Kathleen P. Pratt, PhD, Devi Gunasekera, MBBS, Pooja Vir, PhD, Robert Peters, Siyuan Tan, PhD, Glenn Pierce, MD PhD, Cara Olsen, PhD, Saulius Butenas, PhD, Kenneth G. Mann, PhD

Blood (2019) 134 (Supplement_1): 1131.

https://doi.org/10.1182/blood-2019-124743

Published: 2019

Journal Articles

The Immune Response to Murine Factor VIII in Single Exon Deletion and Total Gene Deletion Murine Models of Hemophilia a

Seema R Patel, PhD, Wallace H. Baldwin, Courtney Cox, MSc, John Healey, BS, Ernest T. Parker, Glaivy Batsuli, MD, Shannon L. Meeks, MD

Blood (2019) 134 (Supplement_1): 1109.

https://doi.org/10.1182/blood-2019-130890

Published: 2019

Journal Articles

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Franziska Horling, PhD, Johannes Lengler, PhD, Wenjing Cao, PhD, Biao Dong, PhD, Bagirath Gangadharan, Maurus De La Rosa, PhD, Werner Hoellriegl, PhD, Birgit M Reipert, PhD, Friedrich Scheiflinger, Weidong Xiao, PhD, Hanspeter Rottensteiner

Blood (2019) 134 (Supplement_1): 3356.

https://doi.org/10.1182/blood-2019-126489

Published: 2019

Journal Articles

Generation of a Unique Cohort of Hemophilia A Dogs Tolerant to Human FVIII for Evaluating the Safety and Efficacy of AAV Delivery of Wild Type and Variant Human FVIII

Giang N. Nguyen, Lauren Wimsey, Elizabeth Merricks, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2018) 132 (Supplement 1): 2453.

https://doi.org/10.1182/blood-2018-99-113492

Published: 2018

Journal Articles

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

Doyoung Lee, PhD, Jenny McIntosh, PhD, Allison Dane, PhD, Cecilia Rosales, PhD, Anne Riddell, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 3484.

https://doi.org/10.1182/blood-2018-99-113830

Published: 2018

Journal Articles

A Novel Mechanism of Factor VIIa/Tissue Factor (TF)-Catalyzed Activation and Inactivation of B-Domain-Deleted Factor VIII in the Early Initiation Phases of Coagulation

Yuto Nakajima, MD, Koji Yada, MD PhD, Keiji Nogami, MDPhD, Midori Shima, MD PhD

Blood (2018) 132 (Supplement 1): 1162.

https://doi.org/10.1182/blood-2018-99-115645

Published: 2018

Journal Articles

The Atlanta Protocol: Immune Tolerance Induction in Pediatric Patients with Hemophilia a and Inhibitors on Emicizumab

Glaivy M. Batsuli, MD, Karen L. Zimowski, MD, Kelly Tickle, MSN, APN, PPCNP-BC, Shannon L. Meeks, MD, Robert F. Sidonio, Jr., MD

Blood (2018) 132 (Supplement 1): 634.

https://doi.org/10.1182/blood-2018-99-114249

Published: 2018

Journal Articles

Potential Long-Term Treatment of Hemophilia a By Early Postnatal Co-Transplantation of Cord Blood Derived Endothelial Colony-Forming Cells and Placental Mesenchymal Stem Cells

Kewa Gao, MD, Priyadarsini Kumar, PhD, Dake Hao, PhD, Lizette Reynaga, BS, Diana Lee Farmer, MD, Melanie Rose, MS, Jan A. Nolta, PhD, Jianda Zhou, MD PhD, Ping Zhou, PhD, Aijun Wang, PhD

Blood (2018) 132 (Supplement 1): 3318.

https://doi.org/10.1182/blood-2018-99-117701

Published: 2018

Journal Articles

A factor VIII–nanobody fusion protein forming an ultrastable complex with VWF: effect on clearance and antibody formation

Vincent Muczynski, Caterina Casari, François Moreau, Gabriel Aymé, Charlotte Kawecki, Paulette Legendre, Valerie Proulle, Olivier D. Christophe, Cécile V. Denis, Peter J. Lenting

Blood (2018) 132 (11): 1193–1197.

https://doi.org/10.1182/blood-2018-01-829523

Published: 2018

Includes: Supplemental data

Journal Articles

Bortezomib-Based Induction Treatment for Patients with Multiple Myeloma Ineligible for Autologous Stem Cell Transplantation: A Retrospective Multicenter Study in Czech Republic

Viera Sandecka, MD, Ludek Pour, Doc,MD PhD, Sabina Sevcikova, RNDr, PhD, Lucie Brozova, MSc, Jan Straub, MD, Vlastimil Scudla, Prof, MD CSc, Vladimir Maisnar, Prof, MD PhD, Petr Pavlicek, MD, Zdenek Adam, Prof, MD CSc, Marta Krejci, Prof, MD PhD, Martin Stork, MD, Tomas Jelinek, Hana Plonkova, Roman Hajek

Blood (2017) 130 (Supplement 1): 5435.

https://doi.org/10.1182/blood.V130.Suppl_1.5435.5435

Published: 2017

Journal Articles

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

Lindsey A. George, MD, Margaret V. Ragni, MD MPH, Ben J. Samelson-Jones, MD PhD, Adam Cuker, MD MS, Alexa R. Runoski, MS, Grace Cole, Fraser Wright, PhD, Yifeng Chen, MD, Daniel J Hui, PhD BS, Katie Wachtel, MS, Daniel Takefman, Linda B. Couto, PhD, Kathleen Z. Reape, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 604.

https://doi.org/10.1182/blood.V130.Suppl_1.604.604

Published: 2017

Journal Articles

Platelet-Targeted Gene Therapy of Hemophilia a Using Hematopoietic Stem Cells Derived from Genome-Edited Induced Pluripotent Stem Cells

Dawei Wang, Guowei Zhang, Junjie Gu, Xiaohu Shao, Xiaohong Pan, Shuxian Yao, Ying Jin, Qizhen Shi, MD PhD, Zhu Chen, PhD, Sai-Juan Chen, PhD

Blood (2017) 130 (Supplement 1): 2063.

https://doi.org/10.1182/blood.V130.Suppl_1.2063.2063

Published: 2017

Journal Articles

Lettuce Plants Expressing High Levels of Factor VIII Antigen in the Chloroplast for Oral Tolerance in Hemophilia a

Roland W Herzog, PhD, Alexandra Sherman, DVM, Kwang-Chul Kwon, PhD, Wan-Jung Chang, MS, Henry Daniell, PhD

Blood (2017) 130 (Supplement 1): 362.

https://doi.org/10.1182/blood.V130.Suppl_1.362.362

Published: 2017

Journal Articles

Comparison of the Functional Characteristics and Post-Translational Modifications of Commercial Factor VIII Products

Guido Kohla, PhD, Larisa Belyanskaya, PhD, Maya Tiemeyer, PhD, Thole Züchner, PhD, Irena Knappik, PhD, Julia Rosenlöcher, PhD

Blood (2017) 130 (Supplement 1): 2362.

https://doi.org/10.1182/blood.V130.Suppl_1.2362.2362

Published: 2017

Journal Articles

FVIII Proteins with a Modified Immunodominant T-Cell Epitope Exhibit Reduced Immunogenicity and Normal Procoagulant Activity

Ruth A. Ettinger, PhD, Joseph A. Liberman, PhD, Devi Gunasekera, BSSM, Eddie A. James, PhD, Arthur R. Thompson, MD PhD, Kathleen P. Pratt, PhD

Blood (2017) 130 (Supplement 1): 179.

https://doi.org/10.1182/blood.V130.Suppl_1.179.179

Published: 2017

Journal Articles

Reprogrammed CD4 + T Cells That Express FoxP3 + Effectively Control Inhibitory Antibody Development in Hemophilic Mice

Moanaro Biswas, PhD, Veronica Kuteyeva, Roland W Herzog, PhD

Blood (2017) 130 (Supplement 1): 175.

https://doi.org/10.1182/blood.V130.Suppl_1.175.175

Published: 2017

Journal Articles

Generation of FVIII-Specific Tregs Expressing a Chimeric Antigen Receptor (CAR) to Suppress Inhibitor Development in Hemophilia a Mice

Moanaro Biswas, PhD, Todd M Brusko, PhD, Roland W Herzog, PhD

Blood (2017) 130 (Supplement 1): 176.

https://doi.org/10.1182/blood.V130.Suppl_1.176.176

Published: 2017

Journal Articles

Structural and Functional Characterization of a Codon Optimized Coagulation Factor VIII

Svetlana A Shestopal, PhD, James H Kurasawa, MS, Jian-Jiang Hao, PhD, Elena Karnaukhova, PhD, Yideng Liang, PhD, Min Lin, PhD, Mikhail V Ovanesov, PhD, Timothy K Lee, PhD, John H McVey, PhD, Andrey G Sarafanov, PhD

Blood (2016) 128 (22): 3765.

https://doi.org/10.1182/blood.V128.22.3765.3765

Published: 2016

NARROW

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Cryo-EM Structure of BIVV001 Reveals Coagulation Factor VIII-Von Willebrand Factor D'D3 Interaction Mode

Neutralizing and Non-Neutralizing Anti-FVIII Antibodies in Black and White Hemophilia A Subjects: A Natural History Profile

The Immune Response to Murine Factor VIII in Single Exon Deletion and Total Gene Deletion Murine Models of Hemophilia a

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Generation of a Unique Cohort of Hemophilia A Dogs Tolerant to Human FVIII for Evaluating the Safety and Efficacy of AAV Delivery of Wild Type and Variant Human FVIII

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

A Novel Mechanism of Factor VIIa/Tissue Factor (TF)-Catalyzed Activation and Inactivation of B-Domain-Deleted Factor VIII in the Early Initiation Phases of Coagulation

The Atlanta Protocol: Immune Tolerance Induction in Pediatric Patients with Hemophilia a and Inhibitors on Emicizumab

Potential Long-Term Treatment of Hemophilia a By Early Postnatal Co-Transplantation of Cord Blood Derived Endothelial Colony-Forming Cells and Placental Mesenchymal Stem Cells

A factor VIII–nanobody fusion protein forming an ultrastable complex with VWF: effect on clearance and antibody formation

Bortezomib-Based Induction Treatment for Patients with Multiple Myeloma Ineligible for Autologous Stem Cell Transplantation: A Retrospective Multicenter Study in Czech Republic

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

Platelet-Targeted Gene Therapy of Hemophilia a Using Hematopoietic Stem Cells Derived from Genome-Edited Induced Pluripotent Stem Cells

Lettuce Plants Expressing High Levels of Factor VIII Antigen in the Chloroplast for Oral Tolerance in Hemophilia a

Comparison of the Functional Characteristics and Post-Translational Modifications of Commercial Factor VIII Products

FVIII Proteins with a Modified Immunodominant T-Cell Epitope Exhibit Reduced Immunogenicity and Normal Procoagulant Activity

Reprogrammed CD4 + T Cells That Express FoxP3 + Effectively Control Inhibitory Antibody Development in Hemophilic Mice

Generation of FVIII-Specific Tregs Expressing a Chimeric Antigen Receptor (CAR) to Suppress Inhibitor Development in Hemophilia a Mice

Structural and Functional Characterization of a Codon Optimized Coagulation Factor VIII

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American Society of Hematology

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