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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

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

In Vivo Model to Evaluate Loss of Liver-Derived Factor IX Expression Caused by AAV Capsid-Specific CD8+ T Cells

David M Markusic, PhD, Ashley T Martino, Ph.D., Federico Mingozzi, PhD., Katherine A. High, MD, Roland W Herzog, PhD

Blood (2012) 120 (21): 2046.

https://doi.org/10.1182/blood.V120.21.2046.2046

Published: 2012

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

Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

Blood (2012) 120 (21): 752.

https://doi.org/10.1182/blood.V120.21.752.752

Published: 2012

Journal Articles

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Blood (2011) 118 (21): 5.

https://doi.org/10.1182/blood.V118.21.5.5

Published: 2011

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

Peptide-Induced Antigen-Specific CD4 + CD25 + FoxP3 + T Cells Suppress Cytotoxicity T Cell Responses Directed Against the AAV Capsid.

Daniel J. Hui, PhD, Etiena Basner-Tschakarjan, MD, Gary C. Pien, MD, PhD, William D. Martin, Annie S. DeGroot, MD, Katherine A. High, MD, Federico Mingozzi, PhD

Blood (2010) 116 (21): 3769.

https://doi.org/10.1182/blood.V116.21.3769.3769

Published: 2010

Journal Articles

Intrinsically Hyperactive and Hyperproliferative CD8 + T Cells In Cmah -/- Mice as a Model of Human Gene Transfer Responses.

George Buchlis, M.S., Federico Mingozzi, PhD., Paula Campos Soto, PhD, Oliver Pearce, PhD, Daniel J. Hui, Ph.D., Ajit P. Varki, MBBS, Katherine A. High, MD

Blood (2010) 116 (21): 3773.

https://doi.org/10.1182/blood.V116.21.3773.3773

Published: 2010

Journal Articles

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Cecilia Rosales, Jenny McIntosh, Anne Riddell, Pradip Rustagi, Bertil Glader, Mark Kay, MD, PhD, James Allay, PhD, John Coleman, Susan Sleep, Katherine A. High, MD, Federico Mingozzi, PhD., John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff

Blood (2010) 116 (21): 248.

https://doi.org/10.1182/blood.V116.21.248.248

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

Suppression of CTL Responses against AAV-Capsid Epitopes by Peptide-Induced Regulatory T Cells.

Daniel J Hui, Ph.D., Gary C Pien, MD, PhD, Etiena Basner-Tschakarjan, M.D., Federico Mingozzi, PhD, Jonathan D Finn, PhD, Shangzhen Zhou, Ph.D., William Martin, Anne S. De Groot, M.D., Katherine A. High, MD

Blood (2009) 114 (22): 377.

https://doi.org/10.1182/blood.V114.22.377.377

Published: 2009

Journal Articles

Proteasome Inhibitors Decrease AAV2 Capsid-Derived Peptide Epitope Presentation On MHC Class I Following Transduction.

Jonathan D Finn, PhD, Daniel J Hui, Ph.D., Downey Harre, Danielle Dunn, Federico Mingozzi, PhD, Shangzhen Zhou, Katherine A. High, MD

Blood (2009) 114 (22): 695.

https://doi.org/10.1182/blood.V114.22.695.695

Published: 2009

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

Blood (2007) 110 (11): 2586.

https://doi.org/10.1182/blood.V110.11.2586.2586

Published: 2007

Journal Articles

Quantifying Capsid Peptide:MHC I Complexes Following Adeno-Associated Virus (AAV) Transduction

Gary C. Pien, MD, PhD, Nicole C. Hasbrouck, MD, Marcela V. Maus, MD, PhD, Federico Mingozzi, PhD, Katherine A. High, MD

Blood (2007) 110 (11): 3737.

https://doi.org/10.1182/blood.V110.11.3737.3737

Published: 2007

Journal Articles

A Novel Splenocyte Approach for Characterizing T Cell Responses to Adeno-Associated Virus in the Normal Population: Implications on Gene Transfer.

Daniel J. Hui, PhD, Federico Mingozzi, PhD, Marcela V. Maus, MD, PhD, Michael A. Tigges, PhD, Glenn F. Pierce, MD, PhD, Katherine A. High, MD

Blood (2006) 108 (11): 3258.

https://doi.org/10.1182/blood.V108.11.3258.3258

Published: 2006

Journal Articles

AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid.

Federico Mingozzi, PhD, Marcela V. Maus, MD,PhD, Denise E. Sabatino, PhD, Daniel J. Hui, PhD, John E.J. Rasko, MBBS, PhD, Margaret V. Ragni, MD, Catherine Scott Manno, MD, Hildegund C.J. Ertl, MD, Katherine A. High, MD

Blood (2006) 108 (11): 455.

https://doi.org/10.1182/blood.V108.11.455.455

Published: 2006

Journal Articles

Characterization of the Immune Response to Canine Factor IX Following AAV-Mediated Intravascular Gene Delivery to Skeletal Muscle in Hemophilia B Dogs.

Daniel J. Hui, PhD, Federico Mingozzi, PhD, Denise E. Sabatino, PhD, Stephanie McCorquodale, Aaron Dillow, Timothy C. Nichols, MD, Valder R. Arruda, MD, PhD., Katherine A. High, MD

Blood (2005) 106 (11): 1297.

https://doi.org/10.1182/blood.V106.11.1297.1297

Published: 2005

NARROW

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

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

In Vivo Model to Evaluate Loss of Liver-Derived Factor IX Expression Caused by AAV Capsid-Specific CD8+ T Cells

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

Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

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

Peptide-Induced Antigen-Specific CD4 + CD25 + FoxP3 + T Cells Suppress Cytotoxicity T Cell Responses Directed Against the AAV Capsid.

Intrinsically Hyperactive and Hyperproliferative CD8 + T Cells In Cmah -/- Mice as a Model of Human Gene Transfer Responses.

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

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

Suppression of CTL Responses against AAV-Capsid Epitopes by Peptide-Induced Regulatory T Cells.

Proteasome Inhibitors Decrease AAV2 Capsid-Derived Peptide Epitope Presentation On MHC Class I Following Transduction.

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

Quantifying Capsid Peptide:MHC I Complexes Following Adeno-Associated Virus (AAV) Transduction

A Novel Splenocyte Approach for Characterizing T Cell Responses to Adeno-Associated Virus in the Normal Population: Implications on Gene Transfer.

AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid.

Characterization of the Immune Response to Canine Factor IX Following AAV-Mediated Intravascular Gene Delivery to Skeletal Muscle in Hemophilia B Dogs.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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