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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

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

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

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

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

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

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

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

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

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

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

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

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