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Ben J. Samelson-Jones
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Journal Articles

rAAV8-PC Gene Therapy for Severe Protein C Deficiency Using a Novel Murine Model

Sarina Levy-Mendelovich, Einat Avishai, Ben J Samelson-Jones, Rima Dardik, Tami barazani-Brutman, Yael Nisgav, Tami Livnat, Gili Kenet
Journal: Blood
Blood (2023) 142 (Supplement 1): 7119.
https://doi.org/10.1182/blood-2023-181140
Published: 2023
Journal Articles

Transfusion-Associated Reversible Cerebral Vasoconstriction Syndrome in Pediatrics: A Case Report and Review of Literature

Saarah Hussain, Ben J Samelson-Jones, Grace Linder
Journal: Blood
Blood (2023) 142 (Supplement 1): 5562.
https://doi.org/10.1182/blood-2023-190503
Published: 2023
Journal Articles

Evolution Directed Gene Engineering (EDGE) Identifies Single Amino Acid Substitutions in FVIII with Six-Fold Increased in Vitro and In Vivo Activity

Lucas Van Gorder, Reilly Peng, Julia Chau, Ben J Samelson-Jones
Journal: Blood
Blood (2023) 142 (Supplement 1): 21.
https://doi.org/10.1182/blood-2023-178106
Published: 2023
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 3628.
https://doi.org/10.1182/blood-2023-179431
Published: 2023
Journal Articles

Factor VIII Mimetic Rescue of Hemophilia B Causing Factor IX Variants

Kyumin Lee, Julia Q Chau, Anna R Sternberg, Allyson M Pishko, Lindsey George, Ben J Samelson-Jones
Journal: Blood
Blood (2022) 140 (Supplement 1): 5570–5571.
https://doi.org/10.1182/blood-2022-164689
Published: 2022
Journal Articles

Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia Α Dog after AAV Gene Therapy

Lucas Van Gorder, Bhavya S. Doshi, Elinor Willis, Matthew Lanza, Valder R. Arruda, Mary Beth Callan, Ben J. Samelson-Jones
Journal: Blood
Blood (2022) 140 (Supplement 1): 4919–4920.
https://doi.org/10.1182/blood-2022-164440
Published: 2022
Journal Articles

Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inbibitors in Privately Owned Dogs

Bhavya S Doshi, Ben J Samelson-Jones, Timothy C Nichols, Elizabeth P Merricks, Valder R Arruda, Mary Beth Callan
Journal: Blood
Blood (2022) 140 (Supplement 1): 7780–7781.
https://doi.org/10.1182/blood-2022-164470
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
Journal: Blood
Blood (2021) 138 (Supplement 1): 3975.
https://doi.org/10.1182/blood-2021-150541
Published: 2021
Journal Articles

Ontogeny of the Alloimmune Anti-Canine Factor VIII Inhibitor Response in Severe Hemophilia Α Dogs

Robert Chen, Bhavya S Doshi, Timothy C. Nichols, Michael C. Milone, Ben J. Samelson-Jones, Valder R Arruda, Vijay Bhoj, Don L. Siegel
Journal: Blood
Blood (2021) 138 (Supplement 1): 3173.
https://doi.org/10.1182/blood-2021-149215
Published: 2021
Journal Articles

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

Katherine A. High, MD, Lindsey A. George, MD, M. Elaine Eyster, MD, Spencer K. Sullivan, MD, Margaret V. Ragni, MD MPH, Stacy E. Croteau, MD, Ben J. Samelson-Jones, MD PhD, Matthew Evans, MD, Marcelyne Joseney-Antoine, Amy Macdougall, BSN, Judith Kadosh, BSN RN, Alexa R. Runoski, MS, Cynthia Campbell-Baird, Kayla Douglas, Summer Tompkins, Howard Hait, Linda B. Couto, PhD, Ashlyn Eaton Bassiri, PhD, Leonard A. Valentino, MD, Marcus E. Carr, MD PhD, Daniel J Hui, PhD BS, Katie Wachtel, MS, Daniel Takefman, Federico Mingozzi, PhD, Xavier M. Anguela, PhD, Kathleen B Reape, MD
Journal: Blood
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 - 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

The Role of Transgene Levels in Immune Tolerance Induction after AAV Gene Therapy in Hemophilia B Canine Models

Ben J. Samelson-Jones, MD PhD, Robert Andrew French, BS, Julie M. Crudele, PhD, Timothy C. Nichols, MD, Valder R Arruda, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 2057.
https://doi.org/10.1182/blood.V130.Suppl_1.2057.2057
Published: 2017
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
Journal: Blood
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
Journal: Blood
Blood (2017) 130 (Supplement 1): 3650.
https://doi.org/10.1182/blood.V130.Suppl_1.3650.3650
Published: 2017
Journal Articles

Factor IX Padua: From Biochemistry to Gene Therapy

Valder Arruda, MD PhD, Ben J. Samelson-Jones, MDPhD
Journal: Blood
Blood (2016) 128 (22): SCI-9.
https://doi.org/10.1182/blood.V128.22.SCI-9.SCI-9
Published: 2016
Journal Articles

New Hyperactive Factor IX Variants Bioengineered Based upon Biochemical Studies of Factor IX Padua

Ben J. Samelson-Jones, MD PhD, Jonathan D Finn, PhD, Rodney M. Camire, PhD, Valder R Arruda, MD PhD
Journal: Blood
Blood (2016) 128 (22): 1384.
https://doi.org/10.1182/blood.V128.22.1384.1384
Published: 2016