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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

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

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

Journal Articles

VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications

Courtney T Connolly, Armida Faella, Timothy C. Nichols, MD, Katherine A. High, MD, Valder R. Arruda, MD, PhD, Paris Margaritis, DPhil

Blood (2014) 124 (21): 1477.

https://doi.org/10.1182/blood.V124.21.1477.1477

Published: 2014

Journal Articles

Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy

Joshua I Siner, Julie M Crudele, Courtney T Connolly, Shangzhen Zhou, MD, Elizabeth P. Merricks, PhD, Timothy C. Nichols, MD, Rodney M. Camire, PhD, Valder R. Arruda, MD PhD

Blood (2014) 124 (21): 105.

https://doi.org/10.1182/blood.V124.21.105.105

Published: 2014

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

Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression

Julie M Crudele, BA, Geerte L. Van Sluis, MD, Paris Margaritis, DPhil, Joshua I Siner, Michael Sliozberg, Jenna Maurer, Armida Faella, Shangzhen Zhou, MD, Katherine A. High, MD, Charles T Esmon, PhD, C. Arnold Spek, PhD, Valder R Arruda, MD, PhD

Blood (2012) 120 (21): 3350.

https://doi.org/10.1182/blood.V120.21.3350.3350

Published: 2012

Journal Articles

Bioengineering Factor VIII B-Domain Sequences Improves Function and Efficacy in Hemophilia A Models.

Joshua I Siner, Nicholas P Iacobelli, Lacramioara Ivanciu, Ph.D., Denise E. Sabatino, Ph.D., Mortimer Poncz, MD, Rodney M. Camire, PhD, Valder R Arruda, MD, PhD

Blood (2012) 120 (21): 2208.

https://doi.org/10.1182/blood.V120.21.2208.2208

Published: 2012

Journal Articles

Platelet Factor VIII-Induced Megakaryocyte Apoptosis: Implications for Hemophilia A Gene Therapy

Teshell K Greene, Ph.D., Li Zhai, MD, Beatrice Razzo, B.S, Karen Vo, Denise E. Sabatino, Ph.D., Rodney M. Camire, PhD, Valder R Arruda, MD, PhD, Mortimer Poncz, MD

Blood (2012) 120 (21): 2051.

https://doi.org/10.1182/blood.V120.21.2051.2051

Published: 2012

Journal Articles

Delivery of a Modified U1 Small Nuclear RNA Alleviates Splicing-Defective Coagulation Factor VII Expression in Mouse Models

Dario Balestra, PhD, Armida Faella, Nicola Cavallari, PhD, Paris Margaritis, PhD, Franco Pagani, MD, Francesco Bernardi, PhD, Valder R Arruda, MD, PhD, Mirko Pinotti, PhD

Blood (2012) 120 (21): 754.

https://doi.org/10.1182/blood.V120.21.754.754

Published: 2012

Journal Articles

Hemophilia Gene Therapy

Valder R. Arruda, MD, PhD

Blood (2011) 118 (21): SCI-48.

https://doi.org/10.1182/blood.V118.21.SCI-48.SCI-48

Published: 2011

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

NARROW

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

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

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

VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications

Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy

Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs

Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression

Bioengineering Factor VIII B-Domain Sequences Improves Function and Efficacy in Hemophilia A Models.

Platelet Factor VIII-Induced Megakaryocyte Apoptosis: Implications for Hemophilia A Gene Therapy

Delivery of a Modified U1 Small Nuclear RNA Alleviates Splicing-Defective Coagulation Factor VII Expression in Mouse Models

Hemophilia Gene Therapy

Rabbit Anti-Thymocyte Globulin (rATG) Administrated Concomitantly with Liver Delivery of AAV2-hFIX Can Promote Inhibitor Formation In Rhesus Macaques.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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