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Journal Articles

Tocilizumab As a Novel Immunomodulatory Regimen for Hemophilia Gene Therapy

Klaudia Kuranda, Christine Weisshaar, Yifeng Chen, Corinne Smith, Heena Beck, Kristen Kahle, Federico Mingozzi

Blood (2021) 138 (Supplement 1): 1853.

https://doi.org/10.1182/blood-2021-147511

Published: 2021

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 Factor IX with Minimal Immune Response

Daniel J Hui, PhD BS, Yun Liu, PhD, Ruchi Patel, Yifeng Chen, MD, Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, John Rasko, MBBS, PhD, Ben J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD, Sue Dasen, MS, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 2056.

https://doi.org/10.1182/blood.V130.Suppl_1.2056.2056

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

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

Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector

Etiena Basner-Tschakarjan, MD PhD, Federico Mingozzi, PhD, Yifeng Chen, MD, Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Anne Riddell, BSc, Catherine Ng, MS, Pradip Rustagi, MD, Bertil Glader, MD, PhD, Mark Kay, MD, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff, MD, Katherine A. High, MD

Blood (2013) 122 (21): 717.

https://doi.org/10.1182/blood.V122.21.717.717

Published: 2013

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

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

NARROW

Tocilizumab As a Novel Immunomodulatory Regimen for Hemophilia Gene Therapy

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 Factor IX with Minimal Immune Response

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

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

Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector

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

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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