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Joshua I. Siner
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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
Journal: Blood
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
Journal: Blood
Blood (2014) 124 (21): 104.
https://doi.org/10.1182/blood.V124.21.104.104
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
Journal: Blood
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
Journal: Blood
Blood (2013) 122 (21): 4203.
https://doi.org/10.1182/blood.V122.21.4203.4203
Published: 2013
Journal Articles

Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

Joshua I. Siner, Nicholas P. Iacobelli, Denise E. Sabatino, Lacramiora Ivanciu, Shangzhen Zhou, Mortimer Poncz, Rodney M. Camire, Valder R. Arruda
Journal: Blood
Blood (2013) 121 (21): 4396–4403.
https://doi.org/10.1182/blood-2012-10-464164
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
Journal: Blood
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
Journal: Blood
Blood (2012) 120 (21): 2208.
https://doi.org/10.1182/blood.V120.21.2208.2208
Published: 2012