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Journal Articles

Characterization of a Novel Human Factor VIIa Chimera with Increased Tissue Factor-Independent Activity for Emergency Hemostasis

Ammon Fager, MD PhD, Maureane Hoffman, MD PhD, Dougald Monroe, PhD

Blood (2019) 134 (Supplement_1): 3623.

https://doi.org/10.1182/blood-2019-124428

Published: 2019

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

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell

Blood (2018) 132 (Supplement 1): 489.

https://doi.org/10.1182/blood-2018-99-118256

Published: 2018

Journal Articles

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek

Blood (2018) 131 (9): 1022–1031.

https://doi.org/10.1182/blood-2017-09-804419

Published: 2018

Includes: Supplemental data

Journal Articles

Phe174-Mutated Human Factor X As Bypassing Agent to the Direct FXa Inhibitors

Mark Schreuder, MD, Daniel Verhoef, MSc, Ka Lei Cheung, Koen Visscher, MSc, Daan Geerke, PhD, Pieter H. Reitsma, PhD, Mettine H.A. Bos, PhD

Blood (2017) 130 (Supplement 1): 363.

https://doi.org/10.1182/blood.V130.Suppl_1.363.363

Published: 2017

Journal Articles

Comparative Glycosylation Mapping of Recombinant and Plasma-Derived Human Factor VIII Reveals Key Biological Differences

Jingyao Qu, PhD, Cheng Ma, PhD, Carol H. Miao, PhD, Peng George Wang, PhD, Weidong Xiao, PhD

Blood (2017) 130 (Supplement 1): 3657.

https://doi.org/10.1182/blood.V130.Suppl_1.3657.3657

Published: 2017

Journal Articles

101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)

Steven Pipe, MD, Kimo Stine, MD, Anita Rajasekhar, MD, Tamara Everington, MD, Allen Poma, MD, Eric Crombez, MD, Charles RM Hay, MD

Blood (2017) 130 (Supplement 1): 3331.

https://doi.org/10.1182/blood.V130.Suppl_1.3331.3331

Published: 2017

Journal Articles

Development of an Optimized rAAV2/6 Human Factor 8 cDNA Vector Cassette for Hemophilia a Gene Therapy

Blood (2016) 128 (22): 1173.

https://doi.org/10.1182/blood.V128.22.1173.1173

Published: 2016

Journal Articles

Novel Human Factor VIII Variant with Impaired Intracellular Processing Exhibits Enhanced In Vivo Efficacy

Giang N. Nguyen, B.S., Christian M. Wood, Tanner Dirstine, B.S., Robert J. Davidson, B.S., Denise E. Sabatino, PhD

Blood (2016) 128 (22): 256.

https://doi.org/10.1182/blood.V128.22.256.256

Published: 2016

Journal Articles

Structural Basis for the Procofactor to Cofactor Transition in Human Factor V

Shekhar Kumar, PhD, Wei Deng, PhD, Steven Stayrook, PhD, Renhao Li, PhD, Rodney M. Camire, PhD, Sriram Krishnaswamy, PhD

Blood (2016) 128 (22): 253.

https://doi.org/10.1182/blood.V128.22.253.253

Published: 2016

Journal Articles

Acquired Factor VIII Inhibitors: A Single Institution Retrospective, Observational Study

Lela Ruck, MD, Morey A. Blinder, MD, William Berger, MSW

Blood (2016) 128 (22): 4971.

https://doi.org/10.1182/blood.V128.22.4971.4971

Published: 2016

Journal Articles

Novel Sites of Gamma Carboxylation in Human Factor VII

Emily M Wilkerson, Barbara Bates, Kraig T Kumfer, Nicholas M Riley, Brad S Schwartz, Joshua J Coon

Blood (2016) 128 (22): 3759.

https://doi.org/10.1182/blood.V128.22.3759.3759

Published: 2016

Journal Articles

The X-Ray Structure of a Variant of Human Factor V Provides Structural Insights into the Procofactor Activation Paradox

Shekhar Kumar, PhD, Steven Stayrook, PhD, Rodney M. Camire, PhD, Sriram Krishnaswamy, PhD

Blood (2015) 126 (23): 121.

https://doi.org/10.1182/blood.V126.23.121.121

Published: 2015

Journal Articles

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Xavier M. Anguela, PhD, Liron Elkouby, PhD, Raffaella Toso, PhD, Marti DiPietro, MS, Robert J. Davidson, Katherine A. High, MD, Denise E. Sabatino, PhD

Blood (2015) 126 (23): 199.

https://doi.org/10.1182/blood.V126.23.199.199

Published: 2015

Journal Articles

Identification of Active Coagulation Factor IX Variants with Insertion or Fusion of Unstructured Polypeptide Xten

Zhiqian Lucy Liu, PhD, Arjan van der Flier, PhD, Sara Bardan, Sheng Ding, Volker Schellenberger, PhD, John Kulman, PhD, Robert Peters, PhD

Blood (2015) 126 (23): 2280.

https://doi.org/10.1182/blood.V126.23.2280.2280

Published: 2015

Journal Articles

Human Factor VIII Expression and Normalization of Bleeding Following AAV Gene Therapy in a Double Knockout Mouse Model of Hemophilia

Stuart Bunting, PhD, Lening Zhang, PhD, Lin Xie, BS, Sherry Bullens, AB, Rajeev Mahimkar, PhD, Sylvia Fong, PhD, Krystal Sandza, BS, Peter Colosi, PhD, Shinong Long, PhD, Gordon Allen Vehar, PhD, Barrie Carter, PhD

Blood (2015) 126 (23): 3239.

https://doi.org/10.1182/blood.V126.23.3239.3239

Published: 2015

Journal Articles

Improved Factor VIII Expression with the Introduction of a PACE-Furin Variant into Codon-Optimized Human Factor VIII

Liron Elkouby, PhD, Robert J. Davidson, Paris Margaritis, DPhil, Katherine A. High, MD, Denise E. Sabatino, PhD

Blood (2014) 124 (21): 3498.

https://doi.org/10.1182/blood.V124.21.3498.3498

Published: 2014

Journal Articles

Surgery in Von Willebrand Disease (VWD) Is Facilitated By a Novel 1:1 Plasma Derived Human Factor VWF/VIII Concentrate

Alok Srivastava, MD, Margit Serban, Prof. Dr., Sylvia Werner, Bruce A. Schwartz, PhD, Sigurd Knaub, PhD, Craig M. Kessler, MD

Blood (2014) 124 (21): 1504.

https://doi.org/10.1182/blood.V124.21.1504.1504

Published: 2014

Journal Articles

Human Factor VIII Variants with Diminished PACE-Furin Cleavage Exhibit Increased Biological Activity and Therapeutic Efficacy

Giang N. Nguyen, Joshua I. Siner, Robert J. Davidson, Valder R. Arruda, MD PhD, Rodney M. Camire, PhD, Denise E. Sabatino, PhD

Blood (2014) 124 (21): 104.

https://doi.org/10.1182/blood.V124.21.104.104

Published: 2014

NARROW

Characterization of a Novel Human Factor VIIa Chimera with Increased Tissue Factor-Independent Activity for Emergency Hemostasis

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

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Phe174-Mutated Human Factor X As Bypassing Agent to the Direct FXa Inhibitors

Comparative Glycosylation Mapping of Recombinant and Plasma-Derived Human Factor VIII Reveals Key Biological Differences

101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)

Development of an Optimized rAAV2/6 Human Factor 8 cDNA Vector Cassette for Hemophilia a Gene Therapy

Novel Human Factor VIII Variant with Impaired Intracellular Processing Exhibits Enhanced In Vivo Efficacy

Structural Basis for the Procofactor to Cofactor Transition in Human Factor V

Acquired Factor VIII Inhibitors: A Single Institution Retrospective, Observational Study

Novel Sites of Gamma Carboxylation in Human Factor VII

The X-Ray Structure of a Variant of Human Factor V Provides Structural Insights into the Procofactor Activation Paradox

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Identification of Active Coagulation Factor IX Variants with Insertion or Fusion of Unstructured Polypeptide Xten

Human Factor VIII Expression and Normalization of Bleeding Following AAV Gene Therapy in a Double Knockout Mouse Model of Hemophilia

Improved Factor VIII Expression with the Introduction of a PACE-Furin Variant into Codon-Optimized Human Factor VIII

Surgery in Von Willebrand Disease (VWD) Is Facilitated By a Novel 1:1 Plasma Derived Human Factor VWF/VIII Concentrate

Human Factor VIII Variants with Diminished PACE-Furin Cleavage Exhibit Increased Biological Activity and Therapeutic Efficacy

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American Society of Hematology

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ASH Publications

American Society of Hematology

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