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Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3347.
https://doi.org/10.1182/blood-2019-124091
Published: 2019
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 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

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
Journal: Blood
Blood (2013) 122 (21): 4210.
https://doi.org/10.1182/blood.V122.21.4210.4210
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

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
Journal: Blood
Blood (2010) 116 (21): 3765.
https://doi.org/10.1182/blood.V116.21.3765.3765
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
Journal: Blood
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
Journal: Blood
Blood (2008) 112 (11): 3542.
https://doi.org/10.1182/blood.V112.11.3542.3542
Published: 2008
Journal Articles

Safety of Recombinant Adeno-Associated Viral Vectors in a Large Animal Model.

Patricia Favaro, PhD, Harre D. Downey, Federico Mingozzi, PhD, Fraser Wright, PhD, Bernd Hauck, PhD, Katherine A. High, MD, Valder R. Arruda, MD, PhD
Journal: Blood
Blood (2007) 110 (11): 2586.
https://doi.org/10.1182/blood.V110.11.2586.2586
Published: 2007
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
Journal: Blood
Blood (2007) 110 (11): 3736.
https://doi.org/10.1182/blood.V110.11.3736.3736
Published: 2007