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Journal Articles

Coagulation factor VIII: biological basis of emerging hemophilia A therapies

Benjamin J. Samelson-Jones, Bhavya S. Doshi, Lindsey A. George

Blood (2024) 144 (21): 2185–2197.

https://doi.org/10.1182/blood.2023023275

Published: 2024

Includes: Supplemental data

Journal Articles

Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab

Kyumin Lee, Julia Q. Chau, Yani B. Suber, Anna R. Sternberg, Allyson M. Pishko, Lindsey A. George, Vijay G. Bhoj, Bhavya S. Doshi, Benjamin J. Samelson-Jones

Blood (2024) 144 (11): 1230–1235.

https://doi.org/10.1182/blood.2023021944

Published: 2024

Includes: Supplemental data

Journal Articles

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Sylvia von Mackensen, Jonathan M. Ducore, Lindsey A. George, Adam Giermasz, Catherine McGuinn, John E. J. Rasko, Ben J Samelson-Jones, Spencer K. Sullivan, Jerome M. Teitel, Amit Chhabra, Annie F. Fang, Amanda O'Brien, Frank Plonski, Jeremy Rupon, Lynne Smith, Ian Winburn

Blood (2023) 142 (Supplement 1): 3628.

https://doi.org/10.1182/blood-2023-179431

Published: 2023

Journal Articles

Factor VIII trafficking to CD4 + T cells shapes its immunogenicity and requires several types of antigen-presenting cells

Blood (2023) 142 (3): 290–305.

https://doi.org/10.1182/blood.2022018937

Published: 2023

Includes: Multimedia, Supplemental data

Journal Articles

Antigen Trafficking and Possibly Intrinsic Immunostimulatory Properties Shape High Immunogenicity of Factor VIII

Radoslaw Kaczmarek, Lindsey A. George, Rodney M. Camire, Roland W Herzog

Blood (2022) 140 (Supplement 1): 967–968.

https://doi.org/10.1182/blood-2022-163879

Published: 2022

Journal Articles

Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia a Gene Therapy

Anna R. Sternberg, Cristina Martos-Rus, Robert J. Davidson, Noelle C. Novak, Amelia R. Wilhelm, Lindsey A. George

Blood (2022) 140 (Supplement 1): 965–966.

https://doi.org/10.1182/blood-2022-170646

Published: 2022

Journal Articles

Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study

Sobenna A George, Anirudh Veludhandi, Yijin Xiang, Elizabeth Stenger, Staci D. Arnold, Akanksha Mehta, David A Schirmer, Jessica B Spencer, Gregory M Guilcher, Monica Bhatia, Allistair Abraham, Veronica Gomez-Lobo, Lakshmanan Krishnamurti, Lillian R Meacham

Blood (2022) 140 (Supplement 1): 1391–1392.

https://doi.org/10.1182/blood-2022-168060

Published: 2022

Journal Articles

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Ben J. Samelson-Jones, Spencer K. Sullivan, John E.J. Rasko, Adam Giermasz, Lindsey A. George, Jonathan M. Ducore, Jerome M. Teitel, Catherine E. McGuinn, Amanda O'Brien, Ian Winburn, Lynne M Smith, Amit Chhabra, Jeremy Rupon

Blood (2021) 138 (Supplement 1): 3975.

https://doi.org/10.1182/blood-2021-150541

Published: 2021

Journal Articles

Activated protein C has a regulatory role in factor VIII function

Amelia R. Wilhelm, Nicole A. Parsons, Benjamin J. Samelson-Jones, Robert J. Davidson, Charles T. Esmon, Rodney M. Camire, Lindsey A. George

Blood (2021) 137 (18): 2532–2543.

https://doi.org/10.1182/blood.2020007562

Published: 2021

Includes: Supplemental data

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

Journal Articles

In Vivo hemostatic Significance of Activated Protein C in Factor VIIIa Regulation

Amelia R. Wilhelm, B.S., Nicole A. Parsons, B.S., Charles T. Esmon, PhD, Rodney M. Camire, PhD, Lindsey A. George, MD

Blood (2019) 134 (Supplement_1): 93.

https://doi.org/10.1182/blood-2019-124493

Published: 2019

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

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

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

Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery

Mary Robinson, PhD, Lindsey A. George, MD, Benjamin J. Samelson-Jones, MD PhD, Valder R Arruda, MD PhD, Katherine A. High, MD, Marcus E Carr, MD PhD, Stefan Tiefenbacher, PhD

Blood (2018) 132 (Supplement 1): 2198.

https://doi.org/10.1182/blood-2018-99-119616

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

Regulation of Factor VIII By Activated Protein C: Assessment In Vitro and In Vivo

Nicole A. Parsons, B.S., Michelle D. Ho, BS, Robert J. Davidson, BS, Nabil K. Thalji, PhDMD, Ben J. Samelson-Jones, MD PhD, Rodney M Camire, PhD, Lindsey A. George, MD

Blood (2017) 130 (Supplement 1): 3650.

https://doi.org/10.1182/blood.V130.Suppl_1.3650.3650

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

Human intrathymic lineage commitment is marked by differential CD7 expression: identification of CD7 − lympho-myeloid thymic progenitors

Qian-Lin Hao, Aswathi A. George, Judy Zhu, Lora Barsky, Ewa Zielinska, Xiuli Wang, Mary Price, Shundi Ge, Gay M. Crooks

Blood (2008) 111 (3): 1318–1326.

https://doi.org/10.1182/blood-2007-08-106294

Published: 2008

Includes: Supplemental data

NARROW

Coagulation factor VIII: biological basis of emerging hemophilia A therapies

Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Factor VIII trafficking to CD4 + T cells shapes its immunogenicity and requires several types of antigen-presenting cells

Antigen Trafficking and Possibly Intrinsic Immunostimulatory Properties Shape High Immunogenicity of Factor VIII

Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia a Gene Therapy

Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Activated protein C has a regulatory role in factor VIII function

No CpGs for AAVs?

In Vivo hemostatic Significance of Activated Protein C in Factor VIIIa Regulation

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

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

Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Regulation of Factor VIII By Activated Protein C: Assessment In Vitro and In Vivo

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

Human intrathymic lineage commitment is marked by differential CD7 expression: identification of CD7 − lympho-myeloid thymic progenitors

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American Society of Hematology

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American Society of Hematology

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