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Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

Blood (2016) 128 (22): 3.

https://doi.org/10.1182/blood.V128.22.3.3

Published: 2016

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

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

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

Assessing the potential for AAV vector genotoxicity in a murine model

Hojun Li, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Giulio Bussadori, Shyrie E. Edmonson, Rachel Shah, Valder R. Arruda, Federico Mingozzi, J. Fraser Wright, Frederic D. Bushman, Katherine A. High

Blood (2011) 117 (12): 3311–3319.

https://doi.org/10.1182/blood-2010-08-302729

Published: 2011

Includes: Supplemental data

Journal Articles

Rabbit Anti-Thymocyte Globulin (rATG) Administrated Concomitantly with Liver Delivery of AAV2-hFIX Can Promote Inhibitor Formation In Rhesus Macaques.

Jonathan D. Finn, PhD, Patricia Favaro, PhD, J. Fraser Wright, PhD, Federico Mingozzi, PhD., Katherine A. High, MD, Valder R. Arruda, MD, PhD

Blood (2010) 116 (21): 3765.

https://doi.org/10.1182/blood.V116.21.3765.3765

Published: 2010

Journal Articles

Correction of ADAMTS13 Deficiency by AAV-Mediated Gene Transfer of a C-Terminal Truncated ADAMTS13 Variant In a Murine Model

Sheng-Yu Jin, M.D., Ph.D., Juan (Jenny) Xiao, M.D., Ph.D., J. Fraser Wright, Ph.D., X. Long Zheng, M.D., Ph.D.

Blood (2010) 116 (21): 251.

https://doi.org/10.1182/blood.V116.21.251.251

Published: 2010

Journal Articles

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Valder R. Arruda, Hansell H. Stedman, Virginia Haurigot, George Buchlis, Stefano Baila, Patricia Favaro, Yifeng Chen, Helen G. Franck, Shangzhen Zhou, J. Fraser Wright, Linda B. Couto, Haiyan Jiang, Glenn F. Pierce, Dwight A. Bellinger, Federico Mingozzi, Timothy C. Nichols, Katherine A. High

Blood (2010) 115 (23): 4678–4688.

https://doi.org/10.1182/blood-2009-12-261156

Published: 2010

Journal Articles

Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice.

Hojun Li, Nirav Malani, Shari R Hamilton, DVM, Alexander Schlachterman, MD, Jim Z Zhang, PhD, Giulio Bussadori, Shyrie A Edmonson, Federico Mingozzi, PhD, J. Fraser Wright, PhD, Valder R. Arruda, MD, PhD, Frederic D Bushman, PhD, Katherine A. High, MD

Blood (2009) 114 (22): 2465.

https://doi.org/10.1182/blood.V114.22.2465.2465

Published: 2009

Journal Articles

Coating of AAV Vectors with Human Albumin Blocks Antibody Binding and Enables Transduction of Human Hepatocytes in the Presence of Neutralizing Antibodies

J. Fraser Wright, PhD

Blood (2008) 112 (11): 3542.

https://doi.org/10.1182/blood.V112.11.3542.3542

Published: 2008

Journal Articles

Trace Capsid DNA Impurities in AAV2 Vectors Are Not Transcribed and Are Unlikely To Contribute to Capsid Peptide-MHC Class I Complexes

Bernd Hauck, Ph.D., Samuel Murphy, Ph.D., Xingge Lui, M.D., Olga Zelenaia, Ph.D., Katherine A. High, M.D., J. Fraser Wright, PhD

Blood (2007) 110 (11): 3736.

https://doi.org/10.1182/blood.V110.11.3736.3736

Published: 2007

Journal Articles

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Federico Mingozzi, Nicole C. Hasbrouck, Etiena Basner-Tschakarjan, Shyrie A. Edmonson, Daniel J. Hui, Denise E. Sabatino, Shangzhen Zhou, J. Fraser Wright, Haiyan Jiang, Glenn F. Pierce, Valder R. Arruda, Katherine A. High

Blood (2007) 110 (7): 2334–2341.

https://doi.org/10.1182/blood-2007-03-080093

Published: 2007

Journal Articles

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII

Ciaran D. Scallan, Tongyao Liu, Amy E. Parker, Susannah L. Patarroyo-White, Haifeng Chen, Haiyan Jiang, Joseph Vargas, Dea Nagy, Sharon K. Powell, J. Fraser Wright, Rita Sarkar, Haig H. Kazazian, Alan McClelland, Linda B. Couto

Blood (2003) 102 (12): 3919–3926.

https://doi.org/10.1182/blood-2003-01-0222

Published: 2003

Journal Articles

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

Ciaran D. Scallan, David Lillicrap, Haiyan Jiang, Xiaobing Qian, Susannah L. Patarroyo-White, Amy E. Parker, Tongyao Liu, Joseph Vargas, Dea Nagy, Sharon K. Powell, J. Fraser Wright, Patricia V. Turner, Shawn J. Tinlin, Sandra E. Webster, Alan McClelland, Linda B. Couto

Blood (2003) 102 (6): 2031–2037.

https://doi.org/10.1182/blood-2003-01-0292

Published: 2003

NARROW

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

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

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

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

Assessing the potential for AAV vector genotoxicity in a murine model

Rabbit Anti-Thymocyte Globulin (rATG) Administrated Concomitantly with Liver Delivery of AAV2-hFIX Can Promote Inhibitor Formation In Rhesus Macaques.

Correction of ADAMTS13 Deficiency by AAV-Mediated Gene Transfer of a C-Terminal Truncated ADAMTS13 Variant In a Murine Model

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice.

Coating of AAV Vectors with Human Albumin Blocks Antibody Binding and Enables Transduction of Human Hepatocytes in the Presence of Neutralizing Antibodies

Trace Capsid DNA Impurities in AAV2 Vectors Are Not Transcribed and Are Unlikely To Contribute to Capsid Peptide-MHC Class I Complexes

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

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American Society of Hematology

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American Society of Hematology

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