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Journal Articles

Second Generation Adeno-Associated Viral Vector Using a Novel Factor VIII Variant with Improved Secretion Achieves Higher Factor VIII Expression in Non-Human Primates

Giang N. Nguyen, Maria C. Seleme, Jonathan R. Lindgren, Allysen C. Henriksen, Amy Muehlmatt, Melanie McFadden, Geary R. Smith, Denise E. Sabatino

Blood (2022) 140 (Supplement 1): 1902–1903.

https://doi.org/10.1182/blood-2022-168340

Published: 2022

Journal Articles

Sustained Factor VIII Activity Following Single Dose of Non-Viral Integrating Gene Therapy

Brian Truong, Giang N. Nguyen, Khal Hajj, Joseph Lucas, Johannes Schwerk, James Yen, Kevin Briseno, Michelle Burrascano, Daniel Smith, Johnnatan Tamayo, Fernando Lopez-Espinoza, Michael Bennett, Blair Madison, Denise E. Sabatino, Joshua Rychak

Blood (2022) 140 (Supplement 1): 969–970.

https://doi.org/10.1182/blood-2022-167919

Published: 2022

Journal Articles

Coagulation Factor VIII Enhances the Cleavage of Von Willebrand Factor By ADAMTS13 In Vivo

Wenjing Cao, Aria Bales, Antonia Bignotti, Noritaka Yada, Liang Zheng, Lindsey George, Denise E. Sabatino, Marie Scully, Rodney M. Camire, X. Long Zheng

Blood (2022) 140 (Supplement 1): 5572–5573.

https://doi.org/10.1182/blood-2022-167539

Published: 2022

Journal Articles

Clogging up the pipeline: factor VIII aggregates

Denise E. Sabatino

Blood (2020) 135 (21): 1825–1827.

https://doi.org/10.1182/blood.2020005450

Published: 2020

Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 611.

https://doi.org/10.1182/blood-2019-126007

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

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

The C Domain Mediates the Export of FVIII from the Endoplasmic Reticulum By Interacting with MCFD2

Zhigang Liu, PhD, Chunlei Zheng, PhD, Denise E. Sabatino, PhD, Bin Zhang, PhD

Blood (2016) 128 (22): 255.

https://doi.org/10.1182/blood.V128.22.255.255

Published: 2016

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

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

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

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

Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

Joshua I. Siner, Nicholas P. Iacobelli, Denise E. Sabatino, Lacramiora Ivanciu, Shangzhen Zhou, Mortimer Poncz, Rodney M. Camire, Valder R. Arruda

Blood (2013) 121 (21): 4396–4403.

https://doi.org/10.1182/blood-2012-10-464164

Published: 2013

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

Journal Articles

Overexpression of Factor VIII Is Associated with Immune Responses to Factor VIII and Cellular Stress in Hemophilia A Mice

Amy M. Lange, B.S., Ekaterina Altynova, B.S., Denise E. Sabatino, Ph.D.

Blood (2011) 118 (21): 22.

https://doi.org/10.1182/blood.V118.21.22.22

Published: 2011

Journal Articles

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Jonathan D. Finn, Margareth C. Ozelo, Denise E. Sabatino, Helen W. G. Franck, Elizabeth P. Merricks, Julie M. Crudele, Shangzhen Zhou, Haig H. Kazazian, David Lillicrap, Timothy C. Nichols, Valder R. Arruda

Blood (2010) 116 (26): 5842–5848.

https://doi.org/10.1182/blood-2010-06-288001

Published: 2010

Journal Articles

Reconstitution of Recombinant Factor VIII In fVIII -/- mice Restores Von Willebrand Factor Homeostasis

Wenjing Cao, M.D., Ph.D., Denise E Sabatino, Ph.D., Ekaterina Altynova, B.S., X. Long Zheng, M.D., Ph.D.

Blood (2010) 116 (21): 2213.

https://doi.org/10.1182/blood.V116.21.2213.2213

Published: 2010

Journal Articles

Understanding Ectopically Expressed Factor VIII (F8) In Megakaryocytes: Implications for Optimum Platelet-Delivered F8 Activity for Gene Therapy

Teshell K Greene, B.S, Denise E. Sabatino, PhD, Nicholas P Iacobelli, B.S, Li Zhai, Steven W. Pipe, MD, M. Anna Kowalska, PhD, Rodney M. Camire, PhD, Valder Arruda, MD, PhD

Blood (2010) 116 (21): 2205.

https://doi.org/10.1182/blood.V116.21.2205.2205

Published: 2010

NARROW

Second Generation Adeno-Associated Viral Vector Using a Novel Factor VIII Variant with Improved Secretion Achieves Higher Factor VIII Expression in Non-Human Primates

Sustained Factor VIII Activity Following Single Dose of Non-Viral Integrating Gene Therapy

Coagulation Factor VIII Enhances the Cleavage of Von Willebrand Factor By ADAMTS13 In Vivo

Clogging up the pipeline: factor VIII aggregates

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

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

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

The C Domain Mediates the Export of FVIII from the Endoplasmic Reticulum By Interacting with MCFD2

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

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

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

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

Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

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

Overexpression of Factor VIII Is Associated with Immune Responses to Factor VIII and Cellular Stress in Hemophilia A Mice

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Reconstitution of Recombinant Factor VIII In fVIII -/- mice Restores Von Willebrand Factor Homeostasis

Understanding Ectopically Expressed Factor VIII (F8) In Megakaryocytes: Implications for Optimum Platelet-Delivered F8 Activity for Gene Therapy

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American Society of Hematology

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ASH Publications

American Society of Hematology

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