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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

Characterization of the Interaction of Activated FVII with the Endothelial Protein C Receptor (EPCR) in the Rat

Juliana Small, PhD, Shannon Zintner, PhD, Lynn E Dankner, Paris Margaritis, DPhil

Blood (2018) 132 (Supplement 1): 2455.

https://doi.org/10.1182/blood-2018-99-116892

Published: 2018

Journal Articles

G-CSF Signaling Counteracts TGF-β—PAI-1-Dependent Retention of Hematopoietic Stem Cells in the Niche

Takashi Yahata, PhD, Abd Aziz Ibrahim, PhD, Toshio Miyata, MD, PhD, Kiyoshi Ando, MD PhD

Blood (2017) 130 (Supplement 1): 1133.

https://doi.org/10.1182/blood.V130.Suppl_1.1133.1133

Published: 2017

Journal Articles

TGF-β–induced intracellular PAI-1 is responsible for retaining hematopoietic stem cells in the niche

Takashi Yahata, Abd Aziz Ibrahim, Yukari Muguruma, Mesut Eren, Alexander M. Shaffer, Nobuo Watanabe, Satoko Kaneko, Tetsuo Nakabayashi, Takashi Dan, Noriaki Hirayama, Douglas E. Vaughan, Toshio Miyata, Kiyoshi Ando

Blood (2017) 130 (21): 2283–2294.

https://doi.org/10.1182/blood-2017-02-767384

Published: 2017

Includes: Supplemental data

Journal Articles

Infusion of Coagulation Factor VIII-Containing Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Shows Potential As Hemophilia a Treatment

Randolph B Lyde, Jr., BS, Hyunsook Ahn, Karen K Vo, BS, Danuta Jadwiga Jarocha, PhD, Li Zhai, BS, Spencer K. Sullivan, MD, Valder R Arruda, MD PhD, Denise E. Sabatino, PhD, Rodney M. Camire, PhD, Deborah French, PhD, Mortimer Poncz, MD

Blood (2016) 128 (22): 2559.

https://doi.org/10.1182/blood.V128.22.2559.2559

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

Manipulating leukocyte interactions in vivo through optogenetic chemokine release

Milka Sarris, Romain Olekhnovitch, Philippe Bousso

Blood (2016) 127 (23): e35–e41.

https://doi.org/10.1182/blood-2015-11-684852

Published: 2016

Includes: Multimedia, Supplemental data

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

The Light Chain of Rat Factor VIII Confers Its Superior Cofactor Activity

Qizhao Wang, PhD, Jenni Firrman, PhD, Katie A Pokiniewski, Wenjing Cao, MD, PhD, Hongying Wei, PhD, Roland Herzog, PhD, Weidong Xiao, PhD

Blood (2015) 126 (23): 2038.

https://doi.org/10.1182/blood.V126.23.2038.2038

Published: 2015

Journal Articles

Towards the Care of Hemophilia a Patients Using Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Expressing Coagulation Factor (F) VIII

Randolph B Lyde, Jr., BS, Li Zhai, BS, Karen Vo, BS, Danuta Jadwiga Jarocha, PhD, Spencer Sullivan, MD, Valder R Arruda, MD PhD, Rodney M. Camire, PhD, Deborah French, PhD, Mortimer Poncz, MD

Blood (2015) 126 (23): 2266.

https://doi.org/10.1182/blood.V126.23.2266.2266

Published: 2015

Journal Articles

Hyperglycemia repression of miR-24 coordinately upregulates endothelial cell expression and secretion of von Willebrand factor

Yaozu Xiang, Jijun Cheng, Dandan Wang, Xiaoyue Hu, Yi Xie, Jeremiah Stitham, Gourg Atteya, Jing Du, Wai Ho Tang, Seung Hee Lee, Kristen Leslie, Geralyn Spollett, Zejian Liu, Erica Herzog, Raimund I. Herzog, Jun Lu, Kathleen A. Martin, John Hwa

Blood (2015) 125 (22): 3377–3387.

https://doi.org/10.1182/blood-2015-01-620278

Published: 2015

Includes: Supplemental data

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

Selectively Targeting Mutated NOTCH1 with a Folate-Thapsigargin Derivative

Giovanni Roti, MD, Jun Qi, PhD, Angela Su, B.S., Antony Varca, B.S., Jon C Aster, MD PhD, James E. Bradner, Kimberly Stegmaier, MD

Blood (2014) 124 (21): 2158.

https://doi.org/10.1182/blood.V124.21.2158.2158

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

Journal Articles

Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy

Joshua I Siner, Julie M Crudele, Courtney T Connolly, Shangzhen Zhou, MD, Elizabeth P. Merricks, PhD, Timothy C. Nichols, MD, Rodney M. Camire, PhD, Valder R. Arruda, MD PhD

Blood (2014) 124 (21): 105.

https://doi.org/10.1182/blood.V124.21.105.105

Published: 2014

Journal Articles

VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications

Courtney T Connolly, Armida Faella, Timothy C. Nichols, MD, Katherine A. High, MD, Valder R. Arruda, MD, PhD, Paris Margaritis, DPhil

Blood (2014) 124 (21): 1477.

https://doi.org/10.1182/blood.V124.21.1477.1477

Published: 2014

Journal Articles

Pharmacokinetics, Efficacy and Safety Of a Recombinant Factor IX (BAX326) In Previously-Treated Pediatric Patients < 12 Years Of Age With Severe Or Moderately Severe Hemophilia B

Tomasz Urasinski, Oleksandra Stasyshyn, Tatiana Andreeva, Luminita Rusen, Farida Perina, MyungShin Oh, Miranda Chapman, Borislava G Pavlova, Wing Yen Wong, Brigitt E. Abbuehl

Blood (2013) 122 (21): 1118.

https://doi.org/10.1182/blood.V122.21.1118.1118

Published: 2013

Journal Articles

Suppression of Inhibitor Formation Against Factor VIII in Hemophilia A Mice by Oral Delivery of Bioencapsulated Antigen

Roland W Herzog, PhD, Dheeraj Verma, PhD, Xiaomei Wang, PhD, Alexandra Sherman, DVM, Shina Lin, BS, Henry Daniell, PhD

Blood (2012) 120 (21): 14.

https://doi.org/10.1182/blood.V120.21.14.14

Published: 2012

Journal Articles

Bioengineering Factor VIII B-Domain Sequences Improves Function and Efficacy in Hemophilia A Models.

Joshua I Siner, Nicholas P Iacobelli, Lacramioara Ivanciu, Ph.D., Denise E. Sabatino, Ph.D., Mortimer Poncz, MD, Rodney M. Camire, PhD, Valder R Arruda, MD, PhD

Blood (2012) 120 (21): 2208.

https://doi.org/10.1182/blood.V120.21.2208.2208

Published: 2012

Journal Articles

Platelet Factor VIII-Induced Megakaryocyte Apoptosis: Implications for Hemophilia A Gene Therapy

Teshell K Greene, Ph.D., Li Zhai, MD, Beatrice Razzo, B.S, Karen Vo, Denise E. Sabatino, Ph.D., Rodney M. Camire, PhD, Valder R Arruda, MD, PhD, Mortimer Poncz, MD

Blood (2012) 120 (21): 2051.

https://doi.org/10.1182/blood.V120.21.2051.2051

Published: 2012

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

Characterization of the Interaction of Activated FVII with the Endothelial Protein C Receptor (EPCR) in the Rat

G-CSF Signaling Counteracts TGF-β—PAI-1-Dependent Retention of Hematopoietic Stem Cells in the Niche

TGF-β–induced intracellular PAI-1 is responsible for retaining hematopoietic stem cells in the niche

Infusion of Coagulation Factor VIII-Containing Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Shows Potential As Hemophilia a Treatment

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

Manipulating leukocyte interactions in vivo through optogenetic chemokine release

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

The Light Chain of Rat Factor VIII Confers Its Superior Cofactor Activity

Towards the Care of Hemophilia a Patients Using Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Expressing Coagulation Factor (F) VIII

Hyperglycemia repression of miR-24 coordinately upregulates endothelial cell expression and secretion of von Willebrand factor

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

Selectively Targeting Mutated NOTCH1 with a Folate-Thapsigargin Derivative

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

Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy

VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications

Pharmacokinetics, Efficacy and Safety Of a Recombinant Factor IX (BAX326) In Previously-Treated Pediatric Patients < 12 Years Of Age With Severe Or Moderately Severe Hemophilia B

Suppression of Inhibitor Formation Against Factor VIII in Hemophilia A Mice by Oral Delivery of Bioencapsulated Antigen

Bioengineering Factor VIII B-Domain Sequences Improves Function and Efficacy in Hemophilia A Models.

Platelet Factor VIII-Induced Megakaryocyte Apoptosis: Implications for Hemophilia A Gene Therapy

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American Society of Hematology

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ASH Publications

American Society of Hematology

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