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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
Journal: Blood
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

Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, Ben J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD, Jerome M. Teitel, MD FRCPC, Adam Cuker, MD MS, Sylvia Von Mackensen, PhD, Suvankar Majumdar, MD, Catherine E. McGuinn, MD, Alexa R. Runoski, MS, Fraser Wright, PhD, Sue Dasen, MS, Kimberly Barber, Yifeng Chen, MD, Daniel J. Hui, PhD, Ruchi Patel, Yun Liu, PhD, Katie Wachtel, MS, Dan Takefman, Linda B. Couto, PhD, Kathleen Z. Reape, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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

Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, PhD, Jonathan M. Ducore, Jerome M. Teitel, MD, Adam Cuker, MD MS, Lisa M. Sullivan, MD, Suvankar Majumdar, MD, Catherine E. McGuinn, MD, Aline M. Galvao, M.S., B.S.N.-R.N., Alvin Y. Luk, PhD, M.B.A., J. Fraser Wright, PhD, Yifeng Chen, MD, Daniel J. Hui, PhD, Katie Wachtel, M.S., Teresa Urich, B.S., Daniel Takefman, PhD, Linda B. Couto, PhD, Marcus E. Carr, MD, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2010) 115 (23): 4678–4688.
https://doi.org/10.1182/blood-2009-12-261156
Published: 2010