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Journal Articles

Factor IXa variants resistant to plasma inhibitors enhance clot formation in vivo

Lacramioara Ivanciu, Valder R. Arruda, Rodney M. Camire

Blood (2023) 141 (16): 2022–2032.

https://doi.org/10.1182/blood.2022018083

Published: 2023

Includes: Multimedia, Supplemental data

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

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

Blood (2022) 140 (Supplement 1): 7780–7781.

https://doi.org/10.1182/blood-2022-164470

Published: 2022

Journal Articles

Immune complications and their management in inherited and acquired bleeding disorders

Valder R. Arruda, David Lillicrap, Roland W. Herzog

Blood (2022) 140 (10): 1075–1085.

https://doi.org/10.1182/blood.2022016530

Published: 2022

Journal Articles

Extra-Splenic Role of B Cell Activating Factor Blockade in Prevention of Factor VIII Inhibitors

Bhavya S Doshi, Mostafa A Shaheen, Juliana C Small, Paris Margaritis, Valder R Arruda

Blood (2021) 138 (Supplement 1): 1025.

https://doi.org/10.1182/blood-2021-149259

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

Blood (2021) 138 (Supplement 1): 3173.

https://doi.org/10.1182/blood-2021-149215

Published: 2021

Journal Articles

The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy

Benjamin J. Samelson-Jones, MD PhD, John D. Finn, PhD, Rodney M. Camire, PhD, Valder R Arruda, MD PhD

Blood (2018) 132 (Supplement 1): 3486.

https://doi.org/10.1182/blood-2018-99-119270

Published: 2018

Journal Articles

Optimized FVIII-Domain-Based Chimeric Antigen Receptors to Specifically Target FVIII Inhibitor-Producing B Cells in Hemophilia a

Vijay Bhoj, MD PhD, Lucy Li, Benjamin J. Samelson-Jones, MD PhD, Bhavya S Doshi, MD, Christoph Ellebrecht, MD, Aimee Payne, MD PhD, Valder R Arruda, MD PhD, Michael C. Milone, MD PhD

Blood (2018) 132 (Supplement 1): 2196.

https://doi.org/10.1182/blood-2018-99-120031

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

The Un-BLyS-Ful State: Blocking Factor VIII Inhibitor Development with BLyS Depletion

Bhavya S Doshi, MD, Robert J. Davidson, BS, Valder R Arruda, MD PhD

Blood (2018) 132 (Supplement 1): 138.

https://doi.org/10.1182/blood-2018-99-114795

Published: 2018

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

Blood (2017) 130 (Supplement 1): 2057.

https://doi.org/10.1182/blood.V130.Suppl_1.2057.2057

Published: 2017

Journal Articles

Novel Correlation between B Cell Survival Cytokines and Inhibitors in Hemophilia A

Bhavya S Doshi, MD, Simon F Lacey, PhD BS, Fang Chen, PhD, Leslie J Raffini, MD MSCE, Valder R Arruda, MD PhD

Blood (2017) 130 (Supplement 1): 3677.

https://doi.org/10.1182/blood.V130.Suppl_1.3677.3677

Published: 2017

Journal Articles

Novel approaches to hemophilia therapy: successes and challenges

Valder R. Arruda, Bhavya S. Doshi, Benjamin J. Samelson-Jones

Blood (2017) 130 (21): 2251–2256.

https://doi.org/10.1182/blood-2017-08-742312

Published: 2017

Journal Articles

Stopping bleeding is not enough to FIX hemarthropathy

Valder R. Arruda

Blood (2017) 129 (15): 2048–2049.

https://doi.org/10.1182/blood-2017-02-760520

Published: 2017

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

Blood (2016) 128 (22): 1384.

https://doi.org/10.1182/blood.V128.22.1384.1384

Published: 2016

Journal Articles

Infusion of Coagulation Factor VIII-Containing Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Shows Potential As Hemophilia a Treatment

Randolph B Lyde, Jr., BS, Hyunsook Ahn, Karen K Vo, BS, Danuta Jadwiga Jarocha, PhD, Li Zhai, BS, Spencer K. Sullivan, MD, Valder R Arruda, MD PhD, Denise E. Sabatino, PhD, Rodney M. Camire, PhD, Deborah French, PhD, Mortimer Poncz, MD

Blood (2016) 128 (22): 2559.

https://doi.org/10.1182/blood.V128.22.2559.2559

Published: 2016

Journal Articles

Towards the Care of Hemophilia a Patients Using Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Expressing Coagulation Factor (F) VIII

Randolph B Lyde, Jr., BS, Li Zhai, BS, Karen Vo, BS, Danuta Jadwiga Jarocha, PhD, Spencer Sullivan, MD, Valder R Arruda, MD PhD, Rodney M. Camire, PhD, Deborah French, PhD, Mortimer Poncz, MD

Blood (2015) 126 (23): 2266.

https://doi.org/10.1182/blood.V126.23.2266.2266

Published: 2015

Journal Articles

Complete Correction of Severe Canine Hemophilia B By Skeletal Muscle Directed AAV-Based FIX-Padua Gene Therapy in Inhibitor-Prone Dogs

Robert French, Nicholas Martin, Timothy C. Nichols, MD, Glenn P Niemeyer, Clinton D. Lothrop, PhD DVM, Valder R Arruda, MD PhD

Blood (2015) 126 (23): 3487.

https://doi.org/10.1182/blood.V126.23.3487.3487

Published: 2015

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

Human Factor VIII Variants with Diminished PACE-Furin Cleavage Exhibit Increased Biological Activity and Therapeutic Efficacy

Giang N. Nguyen, Joshua I. Siner, Robert J. Davidson, Valder R. Arruda, MD PhD, Rodney M. Camire, PhD, Denise E. Sabatino, PhD

Blood (2014) 124 (21): 104.

https://doi.org/10.1182/blood.V124.21.104.104

Published: 2014

NARROW

Factor IXa variants resistant to plasma inhibitors enhance clot formation in vivo

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

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

Immune complications and their management in inherited and acquired bleeding disorders

Extra-Splenic Role of B Cell Activating Factor Blockade in Prevention of Factor VIII Inhibitors

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

The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy

Optimized FVIII-Domain-Based Chimeric Antigen Receptors to Specifically Target FVIII Inhibitor-Producing B Cells in Hemophilia a

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

The Un-BLyS-Ful State: Blocking Factor VIII Inhibitor Development with BLyS Depletion

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

Novel Correlation between B Cell Survival Cytokines and Inhibitors in Hemophilia A

Novel approaches to hemophilia therapy: successes and challenges

Stopping bleeding is not enough to FIX hemarthropathy

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

Infusion of Coagulation Factor VIII-Containing Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Shows Potential As Hemophilia a Treatment

Towards the Care of Hemophilia a Patients Using Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Expressing Coagulation Factor (F) VIII

Complete Correction of Severe Canine Hemophilia B By Skeletal Muscle Directed AAV-Based FIX-Padua Gene Therapy in Inhibitor-Prone Dogs

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

Human Factor VIII Variants with Diminished PACE-Furin Cleavage Exhibit Increased Biological Activity and Therapeutic Efficacy

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American Society of Hematology

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ASH Publications

American Society of Hematology

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