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Journal Articles

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

Blood (2018) 132 (Supplement 1): 487.

https://doi.org/10.1182/blood-2018-99-115495

Published: 2018

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Blood (2017) 130 (Supplement 1): 601.

https://doi.org/10.1182/blood.V130.Suppl_1.601.601

Published: 2017

Journal Articles

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

Lindsey A. George, MD, Margaret V. Ragni, MD MPH, Ben J. Samelson-Jones, MD PhD, Adam Cuker, MD MS, Alexa R. Runoski, MS, Grace Cole, Fraser Wright, PhD, Yifeng Chen, MD, Daniel J Hui, PhD BS, Katie Wachtel, MS, Daniel Takefman, Linda B. Couto, PhD, Kathleen Z. Reape, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 604.

https://doi.org/10.1182/blood.V130.Suppl_1.604.604

Published: 2017

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

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

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Haiyan Jiang, Linda B. Couto, Susannah Patarroyo-White, Tongyao Liu, Dea Nagy, Joseph A. Vargas, Shangzhen Zhou, Ciaran D. Scallan, Jurg Sommer, Sharmila Vijay, Federico Mingozzi, Katherine A. High, Glenn F. Pierce

Blood (2006) 108 (10): 3321–3328.

https://doi.org/10.1182/blood-2006-04-017913

Published: 2006

Journal Articles

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Haiyan Jiang, David Lillicrap, Susannah Patarroyo-White, Tongyao Liu, Xiaobing Qian, Ciaran D. Scallan, Sandra Powell, Tracey Keller, Morag McMurray, Andrea Labelle, Dea Nagy, Joseph A. Vargas, Shangzhen Zhou, Linda B. Couto, Glenn F. Pierce

Blood (2006) 108 (1): 107–115.

https://doi.org/10.1182/blood-2005-12-5115

Published: 2006

Journal Articles

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

Ciaran D. Scallan, Haiyan Jiang, Tongyao Liu, Susannah Patarroyo-White, Jurg M. Sommer, Shangzhen Zhou, Linda B. Couto, Glenn F. Pierce

Blood (2006) 107 (5): 1810–1817.

https://doi.org/10.1182/blood-2005-08-3229

Published: 2006

Journal Articles

AAV8-Factor IX Hepatic Gene Transfer with Transient Immunosuppression Is Safe but Is Abrogated by Low Titers of Neutralizing Antibody in Rhesus Macaques.

Haiyan Jiang, PhD, Susannah Patarroyo-White, BS, Tongyao Liu, BS, Bin Yang, Ph.D, Dea Nagy, M.D, Lin He, M.D, Ciaran D. Scallan, Ph.D, Jurg Sommer, Ph.D, Shangzhen Zhou, M.D, Linda B. Couto, Ph.D, Katherine A. High, M.D, Glenn F. Pierce, M.D/Ph.D

Blood (2005) 106 (11): 5532.

https://doi.org/10.1182/blood.V106.11.5532.5532

Published: 2005

Journal Articles

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Valder R. Arruda, Hansell H. Stedman, Timothy C. Nichols, Mark E. Haskins, Matthew Nicholson, Roland W. Herzog, Linda B. Couto, Katherine A. High

Blood (2005) 105 (9): 3458–3464.

https://doi.org/10.1182/blood-2004-07-2908

Published: 2005

Journal Articles

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1

Valder R. Arruda, Joerg Schuettrumpf, Roland W. Herzog, Timothy C. Nichols, Nancy Robinson, Yasmin Lotfi, Federico Mingozzi, Weidong Xiao, Linda B. Couto, Katherine A. High

Blood (2004) 103 (1): 85–92.

https://doi.org/10.1182/blood-2003-05-1446

Published: 2004

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

Journal Articles

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Blood (2003) 101 (8): 2963–2972.

https://doi.org/10.1182/blood-2002-10-3296

Published: 2003

Journal Articles

Posttranslational modifications of recombinant myotube-synthesized human factor IX

Valder R. Arruda, James N. Hagstrom, Jeffrey Deitch, Terry Heiman-Patterson, Rodney M. Camire, Kirk Chu, Paul A. Fields, Roland W. Herzog, Linda B. Couto, Peter J. Larson, Katherine A. High

Blood (2001) 97 (1): 130–138.

https://doi.org/10.1182/blood.V97.1.130

Published: 2001

Journal Articles

Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

J. Nathan Hagstrom, Linda B. Couto, Ciaran Scallan, Melissa Burton, Mark L. McCleland, Paul A. Fields, Valder R. Arruda, Roland W. Herzog, Katherine A. High

Blood (2000) 95 (8): 2536–2542.

https://doi.org/10.1182/blood.V95.8.2536

Published: 2000

Journal Articles

Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver

Hiroyuki Nakai, Roland W. Herzog, J. Nathan Hagstrom, Johannes Walter, Szu-Hao Kung, Edmund Y. Yang, Shing Jen Tai, Yuichi Iwaki, Gary J. Kurtzman, Krishna J. Fisher, Peter Colosi, Linda B. Couto, Katherine A. High

Blood (1998) 91 (12): 4600–4607.

https://doi.org/10.1182/blood.V91.12.4600

Published: 1998

NARROW

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

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

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

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

AAV8-Factor IX Hepatic Gene Transfer with Transient Immunosuppression Is Safe but Is Abrogated by Low Titers of Neutralizing Antibody in Rhesus Macaques.

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1

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

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Posttranslational modifications of recombinant myotube-synthesized human factor IX

Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter

Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver

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American Society of Hematology

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American Society of Hematology

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