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Journal Articles

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

Oscar A. Marcos-Contreras, Shannon M. Smith, Dwight A. Bellinger, Robin A. Raymer, Elizabeth Merricks, Armida Faella, Giulia Pavani, Shangzhen Zhou, Timothy C. Nichols, Katherine A. High, Paris Margaritis

Blood (2016) 127 (5): 565–571.

https://doi.org/10.1182/blood-2015-09-671420

Published: 2016

Includes: Supplemental data

Journal Articles

In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair

Xavier M. Anguela, PhD, Rajiv Sharma, PhD, Yannick Doyon, PhD, Thomas Wechsler, PhD, David Paschon, PhD, Robert J. Davidson, Shangzhen Zhou, MD, Philip D Gregory, PhD, Michael C. Holmes, PhD, Katherine A. High, MD

Blood (2015) 126 (23): 4422.

https://doi.org/10.1182/blood.V126.23.4422.4422

Published: 2015

Journal Articles

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Thomas Wechsler, Russell C. DeKelver, Scott Sproul, David E. Paschon, Jeffrey C. Miller, Robert J. Davidson, David Shivak, Shangzhen Zhou, Julianne Rieders, Philip D. Gregory, Michael C. Holmes, Edward J. Rebar, Katherine A. High

Blood (2015) 126 (15): 1777–1784.

https://doi.org/10.1182/blood-2014-12-615492

Published: 2015

Includes: Supplemental data

Journal Articles

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Julie M. Crudele, Jonathan D. Finn, Joshua I. Siner, Nicholas B. Martin, Glenn P. Niemeyer, Shangzhen Zhou, Federico Mingozzi, Clinton D. Lothrop, Jr, Valder R. Arruda

Blood (2015) 125 (10): 1553–1561.

https://doi.org/10.1182/blood-2014-07-588194

Published: 2015

Includes: Supplemental data

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

Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs

Julie M Crudele, Jonathan D Finn, PhD, Nicholas B Martin, Joshua I Siner, Yifeng Chen, MD, Shangzhen Zhou, MD, Glenn Niemeyer, PhD, Clinton D. Lothrop, PhD, DVM, Federico Mingozzi, PhD., Katherine A. High, MD, Valder R. Arruda, MD, PhD

Blood (2013) 122 (21): 4203.

https://doi.org/10.1182/blood.V122.21.4203.4203

Published: 2013

Journal Articles

Bioengineered Coagulation Factor VIII Enables Long-Term Correction Of Murine Hemophilia A Following Liver-Directed Adeno-Associated Viral Vector Delivery

Harrison C. Brown, BBA, Jordan E Shields, Shangzhen Zhou, MD, J. Fraser Wright, PhD, H. Trent Spencer, PhD, Christopher B Doering, PhD

Blood (2013) 122 (21): 4210.

https://doi.org/10.1182/blood.V122.21.4210.4210

Published: 2013

Journal Articles

ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A

Xavier M Anguela, PhD, Rajiv Sharma, Yannick Doyon, PhD, Thomas Wechsler, PhD, David E Paschon, PhD, Robert J Davidson, Shangzhen Zhou, MD, Philip D Gregory, PhD, Michael C. Holmes, PhD, Edward J Rebar, PhD, Katherine A. High, MD

Blood (2013) 122 (21): 720.

https://doi.org/10.1182/blood.V122.21.720.720

Published: 2013

Journal Articles

Robust ZFN-mediated genome editing in adult hemophilic mice

Xavier M. Anguela, Rajiv Sharma, Yannick Doyon, Jeffrey C. Miller, Hojun Li, Virginia Haurigot, Michelle E. Rohde, Sunnie Y. Wong, Robert J. Davidson, Shangzhen Zhou, Philip D. Gregory, Michael C. Holmes, Katherine A. High

Blood (2013) 122 (19): 3283–3287.

https://doi.org/10.1182/blood-2013-04-497354

Published: 2013

Includes: Supplemental data

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

AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura

Sheng-Yu Jin, Juan Xiao, Jialing Bao, Shangzhen Zhou, J. Fraser Wright, X. Long Zheng

Blood (2013) 121 (19): 3825–3829.

https://doi.org/10.1182/blood-2013-02-486779

Published: 2013

Includes: Supplemental data

Journal Articles

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8 + T cells

Ashley T. Martino, Etiena Basner-Tschakarjan, David M. Markusic, Jonathan D. Finn, Christian Hinderer, Shangzhen Zhou, David A. Ostrov, Arun Srivastava, Hildegund C. J. Ertl, Cox Terhorst, Katherine A. High, Federico Mingozzi, Roland W. Herzog

Blood (2013) 121 (12): 2224–2233.

https://doi.org/10.1182/blood-2012-10-460733

Published: 2013

Includes: Supplemental data

Journal Articles

Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression

Julie M Crudele, BA, Geerte L. Van Sluis, MD, Paris Margaritis, DPhil, Joshua I Siner, Michael Sliozberg, Jenna Maurer, Armida Faella, Shangzhen Zhou, MD, Katherine A. High, MD, Charles T Esmon, PhD, C. Arnold Spek, PhD, Valder R Arruda, MD, PhD

Blood (2012) 120 (21): 3350.

https://doi.org/10.1182/blood.V120.21.3350.3350

Published: 2012

Journal Articles

A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV

Federico Mingozzi, PhD, Xavier M Anguela, PhD, Giulia Pavani, MS, Yifeng Chen, MD, Robert J Davidson, Daniel J. Hui, Ph.D., Christian J Hinderer, MS, Armida Faella, Crolann Howard, BS, Alex Tai, MS, Gregory M Podsakoff, Margaret V. Ragni, MD, MPH, Shangzhen Zhou, MD, Etiena Basner-Tschakarjan, J. Fraser Wright, PhD, Katherine A. High, MD

Blood (2012) 120 (21): 2050.

https://doi.org/10.1182/blood.V120.21.2050.2050

Published: 2012

Journal Articles

In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9

Xavier M Anguela, PhD, Rajiv Sharma, Yannick Doyon, PhD, Sunnie Y Wong, David E Paschon, Hojun Li, MD PhD, Virginia Haurigot, PhD, Robert J Davidson, Shangzhen Zhou, MD, Philip D. Gregory, PhD, Michael C. Holmes, PhD, Edward Rebar, PhD, Katherine A. High, MD

Blood (2012) 120 (21): 751.

https://doi.org/10.1182/blood.V120.21.751.751

Published: 2012

Journal Articles

Robust Factor IX Expression Following ZFN-Mediated Genome Editing in An Adult Mouse Model of Hemophilia B

Xavier M Anguela, Rajiv Sharma, Hojun Li, Virginia Haurigot, Anand Bhagwat, Robert J Davidson, Shangzhen Zhou, Yannick Doyon, Philip D. Gregory, PhD, Michael C. Holmes, PhD, Katherine A. High, MD

Blood (2011) 118 (21): 668.

https://doi.org/10.1182/blood.V118.21.668.668

Published: 2011

Journal Articles

Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo

Paris Margaritis, Elise Roy, Armida Faella, Harre D. Downey, Lacramioara Ivanciu, Giulia Pavani, Shangzhen Zhou, Ralph M. Bunte, Katherine A. High

Blood (2011) 117 (15): 3974–3982.

https://doi.org/10.1182/blood-2010-09-309732

Published: 2011

Includes: Supplemental data

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

Phenotypic Correction of a Mouse Model of Hemophilia B by In Vivo Genetic Correction of the F9 Gene

Hojun Li, Virginia Haurigot, Yannick Doyon, James Li, Anand Bhagwat, Sunnie Wong, Xavier Anguela, Rajiv Sharma, Lacramioara Ivanciu, Samuel Murphy, Shangzhen Zhou, David Paschon, Edward Rebar, Philip D. Gregory, PhD, Michael C. Holmes, Katherine A. High, MD

Blood (2010) 116 (21): LBA-5.

https://doi.org/10.1182/blood.V116.21.LBA-5.LBA-5

Published: 2010

Journal Articles

Zymogen Protein C as a Novel Modulator of Cancer Progression In Murine Models

Geerte L. Van Sluis, Paris Margaritis, DPhil, Michael Sliozberg, Jenna Mauer, Armida Faella, Shangzhen Zhou, Katherine A. High, MD, Arnold Spek, Valder R. Arruda, MD, PhD

Blood (2010) 116 (21): 718.

https://doi.org/10.1182/blood.V116.21.718.718

Published: 2010

NARROW

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

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

Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs

Bioengineered Coagulation Factor VIII Enables Long-Term Correction Of Murine Hemophilia A Following Liver-Directed Adeno-Associated Viral Vector Delivery

ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A

Robust ZFN-mediated genome editing in adult hemophilic mice

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

AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8 + T cells

Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression

A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV

In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9

Robust Factor IX Expression Following ZFN-Mediated Genome Editing in An Adult Mouse Model of Hemophilia B

Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo

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

Phenotypic Correction of a Mouse Model of Hemophilia B by In Vivo Genetic Correction of the F9 Gene

Zymogen Protein C as a Novel Modulator of Cancer Progression In Murine Models

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American Society of Hematology

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American Society of Hematology

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