Page 1 | Search Results | Blood | American Society of Hematology

1-17 of 17

Sort by

Sort Order Select

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

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 - 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 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

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

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

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

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

Federico Mingozzi, Janneke J. Meulenberg, Daniel J. Hui, Etiena Basner-Tschakarjan, Nicole C. Hasbrouck, Shyrie A. Edmonson, Natalie A. Hutnick, Michael R. Betts, John J. Kastelein, Erik S. Stroes, Katherine A. High

Blood (2009) 114 (10): 2077–2086.

https://doi.org/10.1182/blood-2008-07-167510

Published: 2009

Includes: Supplemental data

Journal Articles

Immunosuppression Modulates Immune Responses to AAV Capsid in Human Subjects Undergoing Intramuscular Gene Transfer for Lipoprotein Lipase Deficiency

Daniel J Hui, Ph.D., Federico Mingozzi, Ph.D., Annemarie Kleefstra, Janneke M Meulenberg, Ph.D., Shyrie Edmonson, Denise Morin, Daniel Gaudet, M.D., Ph.D., Sander van Deventer, M.D., Ph.D., Katherine A. High, M.D.

Blood (2008) 112 (11): 822.

https://doi.org/10.1182/blood.V112.11.822.822

Published: 2008

Journal Articles

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Federico Mingozzi, Nicole C. Hasbrouck, Etiena Basner-Tschakarjan, Shyrie A. Edmonson, Daniel J. Hui, Denise E. Sabatino, Shangzhen Zhou, J. Fraser Wright, Haiyan Jiang, Glenn F. Pierce, Valder R. Arruda, Katherine A. High

Blood (2007) 110 (7): 2334–2341.

https://doi.org/10.1182/blood-2007-03-080093

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

Conserved Amino Acid Sequences in Parvovirus B19 and Adeno-Associated Virus Stimulate Functionally Cross-Reactive CD8 T Cells.

Samuel L. Murphy, PhD, Daniel J. Hui, PhD, Shyrie Edmonson, Katherine A. High, MD

Blood (2006) 108 (11): 3269.

https://doi.org/10.1182/blood.V108.11.3269.3269

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

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 - 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 Mean Factor IX Activity Levels of >30% without Immunosuppression

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

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.

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.

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

Immunosuppression Modulates Immune Responses to AAV Capsid in Human Subjects Undergoing Intramuscular Gene Transfer for Lipoprotein Lipase Deficiency

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

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

Conserved Amino Acid Sequences in Parvovirus B19 and Adeno-Associated Virus Stimulate Functionally Cross-Reactive CD8 T Cells.

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.

Email alerts

ASH Publications

American Society of Hematology

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only