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Journal Articles

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Blood (2022) 140 (Supplement 1): 1899–1901.

https://doi.org/10.1182/blood-2022-158903

Published: 2022

Journal Articles

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

Huyen Tran, M. Elaine Eyster, Stacy E. Croteau, Margaret V. Ragni, Benjamin J. Samelson-Jones, Spencer Sullivan, John E.J. Rasko, Kristen Jaworski, Amy MacDougall, Savina Jaeger, Magdalena Hofer, Charlie Li, Federico Mingozzi, Tiffany Chang, Gallia Levy

Blood (2022) 140 (Supplement 1): 10662–10664.

https://doi.org/10.1182/blood-2022-159027

Published: 2022

Journal Articles

The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011

Matthew S. Evans, Witold B. Rybka, Stacy E. Croteau, Huyen Tran, John E.J. Rasko, Kristen Jaworski, Amy MacDougall, Savina Jaeger, Federico Mingozzi, Tiffany Chang, Gallia Levy

Blood (2022) 140 (Supplement 1): 10654–10655.

https://doi.org/10.1182/blood-2022-159017

Published: 2022

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

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

Interim Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in a Phase-1/2 Clinical Study of Bmn 270, an AAV5-Mediated Gene Therapy for Hemophilia a

Brian Long, PhD MS, BSMedTech, Benjamin Kim, Wing Yen Wong, Ke Yang, Christian Vettermann, Nancy Pryer, Romain Hardet, Klaudia Kuranda, Philippe Veron, Federico Mingozzi, Glenn F. Pierce, Becky Schweighardt

Blood (2017) 130 (Supplement 1): 4611.

https://doi.org/10.1182/blood.V130.Suppl_1.4611.4611

Published: 2017

Journal Articles

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Julie M. Crudele, Jonathan D. Finn, Joshua I. Siner, Nicholas B. Martin, Glenn P. Niemeyer, Shangzhen Zhou, Federico Mingozzi, Clinton D. Lothrop, Jr, Valder R. Arruda

Blood (2015) 125 (10): 1553–1561.

https://doi.org/10.1182/blood-2014-07-588194

Published: 2015

Includes: Supplemental data

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

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Federico Mingozzi, Katherine A. High

Blood (2013) 122 (1): 23–36.

https://doi.org/10.1182/blood-2013-01-306647

Published: 2013

Journal Articles

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Blood (2013) 121 (17): 3335–3344.

https://doi.org/10.1182/blood-2012-10-462200

Published: 2013

Includes: Supplemental data

Journal Articles

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8 + T cells

Ashley T. Martino, Etiena Basner-Tschakarjan, David M. Markusic, Jonathan D. Finn, Christian Hinderer, Shangzhen Zhou, David A. Ostrov, Arun Srivastava, Hildegund C. J. Ertl, Cox Terhorst, Katherine A. High, Federico Mingozzi, Roland W. Herzog

Blood (2013) 121 (12): 2224–2233.

https://doi.org/10.1182/blood-2012-10-460733

Published: 2013

Includes: Supplemental data

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

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

George Buchlis, Gregory M. Podsakoff, Antonetta Radu, Sarah M. Hawk, Alan W. Flake, Federico Mingozzi, Katherine A. High

Blood (2012) 119 (13): 3038–3041.

https://doi.org/10.1182/blood-2011-09-382317

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

Assessing the potential for AAV vector genotoxicity in a murine model

Hojun Li, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Giulio Bussadori, Shyrie E. Edmonson, Rachel Shah, Valder R. Arruda, Federico Mingozzi, J. Fraser Wright, Frederic D. Bushman, Katherine A. High

Blood (2011) 117 (12): 3311–3319.

https://doi.org/10.1182/blood-2010-08-302729

Published: 2011

Includes: Supplemental data

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

NARROW

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

The Effects of Immunomodulation with Corticosteroids to Manage an AAV Capsid Immune response in the Phase I/II Study of SPK-8011

Tocilizumab As a Novel Immunomodulatory Regimen for Hemophilia Gene Therapy

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

Interim Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in a Phase-1/2 Clinical Study of Bmn 270, an AAV5-Mediated Gene Therapy for Hemophilia a

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

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

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8 + T cells

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

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Assessing the potential for AAV vector genotoxicity in a murine model

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.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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