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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

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

Journal Articles

Induction of Immune Tolerance to Canine FVIII in Hemophilia a Dogs with Inhibitors Using AAV-Mediated Expression of Canine FVIII

Jonathan D Finn, PhD, Denise E Sabatino, PhD, Margareth C Ozelo, MD, Shangzhen Zhou, MD, David Lillicrap, MD, Haig H Kazazian, MD, Timothy C. Nichols, MD, Valder R. Arruda, M.D., Ph.D.

Blood (2008) 112 (11): 243.

https://doi.org/10.1182/blood.V112.11.243.243

Published: 2008

Journal Articles

Successful Production of Canine FVIII: Biochemical and Functional Characterization in Hemophilia A Dogs.

Christian Furlan Freguia, PhD, Denise E. Sabatino, PhD, Andrey Santos, BS, Amy Lange, BS, Raffaella Toso, PhD, Timothy C. Nichols, MD, Rodney M. Camire, PhD, Valder R. Arruda, MD, PhD

Blood (2007) 110 (11): 495.

https://doi.org/10.1182/blood.V110.11.495.495

Published: 2007

Journal Articles

AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid.

Federico Mingozzi, PhD, Marcela V. Maus, MD,PhD, Denise E. Sabatino, PhD, Daniel J. Hui, PhD, John E.J. Rasko, MBBS, PhD, Margaret V. Ragni, MD, Catherine Scott Manno, MD, Hildegund C.J. Ertl, MD, Katherine A. High, MD

Blood (2006) 108 (11): 455.

https://doi.org/10.1182/blood.V108.11.455.455

Published: 2006

Journal Articles

Long Term Dose-Dependent Correction of Hemophilia A Dogs Using AAV-8 and AAV-9-Mediated FVIII Gene Transfer.

Denise E. Sabatino, PhD, Amy M. Lange, Melinda Mucci, Rita Sarkar, PhD, Aaron M. Dillow, Timothy C. Nichols, MD, Valder R. Arruda, MD, Haig H. Kazazian, MD

Blood (2006) 108 (11): 999.

https://doi.org/10.1182/blood.V108.11.999.999

Published: 2006

Journal Articles

Characterization of the Immune Response to Canine Factor IX Following AAV-Mediated Intravascular Gene Delivery to Skeletal Muscle in Hemophilia B Dogs.

Daniel J. Hui, PhD, Federico Mingozzi, PhD, Denise E. Sabatino, PhD, Stephanie McCorquodale, Aaron Dillow, Timothy C. Nichols, MD, Valder R. Arruda, MD, PhD., Katherine A. High, MD

Blood (2005) 106 (11): 1297.

https://doi.org/10.1182/blood.V106.11.1297.1297

Published: 2005

NARROW

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

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

Induction of Immune Tolerance to Canine FVIII in Hemophilia a Dogs with Inhibitors Using AAV-Mediated Expression of Canine FVIII

Successful Production of Canine FVIII: Biochemical and Functional Characterization in Hemophilia A Dogs.

AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid.

Long Term Dose-Dependent Correction of Hemophilia A Dogs Using AAV-8 and AAV-9-Mediated FVIII Gene Transfer.

Characterization of the Immune Response to Canine Factor IX Following AAV-Mediated Intravascular Gene Delivery to Skeletal Muscle in Hemophilia B Dogs.

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American Society of Hematology

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American Society of Hematology

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