Search Results - "Siner, Joshua I"

Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype by Siner, Joshua I., Iacobelli, Nicholas P., Sabatino, Denise E., Ivanciu, Lacramiora, Zhou, Shangzhen, Poncz, Mortimer, Camire, Rodney M., Arruda, Valder R.

“…Recombinant canine B-domain deleted (BDD) factor VIII (FVIII) is predominantly expressed as a single-chain protein and exhibits greater stability after…”
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Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model by Favaro, Patricia, Finn, Jonathan D, Siner, Joshua I, Wright, J Fraser, High, Katherine A, Arruda, Valder R

“…Intravascular delivery of adeno-associated virus (AAV) vector is commonly used for liver-directed gene therapy. In humans, the high prevalence of neutralizing…”
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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice by Crudele, Julie M., Finn, Jonathan D., Siner, Joshua I., Martin, Nicholas B., Niemeyer, Glenn P., Zhou, Shangzhen, Mingozzi, Federico, Lothrop, Clinton D., Arruda, Valder R.

“…Emerging successful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk of cellular immune…”
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Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models by Siner, Joshua I, Samelson-Jones, Benjamin J, Crudele, Julie M, French, Robert A, Lee, Benjamin J, Zhou, Shanzhen, Merricks, Elizabeth, Raymer, Robin, Nichols, Timothy C, Camire, Rodney M, Arruda, Valder R

“…Processing by the proprotein convertase furin is believed to be critical for the biological activity of multiple proteins involved in hemostasis, including…”
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AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life by Doshi, Bhavya S., Samelson-Jones, Benjamin J., Nichols, Timothy C., Merricks, Elizabeth P., Siner, Joshua I., French, Robert A., Lee, Ben J., Arruda, Valder R., Callan, Mary Beth

“…The hemophilias are the most common severe inherited bleeding disorders and are caused by deficiency of clotting factor (F) VIII (hemophilia A) or FIX…”
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Human Factor VIII Variants with Diminished PACE-Furin Cleavage Exhibit Increased Biological Activity and Therapeutic Efficacy by Nguyen, Giang N., Siner, Joshua I., Davidson, Robert J., Arruda, Valder R., Camire, Rodney M., Sabatino, Denise E.

“…During intracellular processing, factor VIII (FVIII) undergoes proteolysis at multiple sites with the most predominant cleavage at a Paired basic Amino acid…”
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Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy by Siner, Joshua I, Crudele, Julie M, Connolly, Courtney T, Zhou, Shangzhen, Merricks, Elizabeth P., Nichols, Timothy C., Camire, Rodney M., Arruda, Valder R.

“…The paired basic amino acid cleaving enzyme (PACE)/Furin is a protein convertase system that plays a vital role in several biological processes, including…”
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Bioengineering Factor VIII B-Domain Sequences Improves Function and Efficacy in Hemophilia A Models by Siner, Joshua I, Iacobelli, Nicholas P, Ivanciu, Lacramioara, Sabatino, Denise E., Poncz, Mortimer, Camire, Rodney M., Arruda, Valder R

“…Abstract 2208 We previously reported that recombinant canine B-domain deleted factor VIII (FVIII) was expressed predominantly (>75%) as a single-chain protein…”
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Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs by Crudele, Julie M, Finn, Jonathan D, Martin, Nicholas B, Siner, Joshua I, Chen, Yifeng, Zhou, Shangzhen, Niemeyer, Glenn, Lothrop, Clinton D., Mingozzi, Federico, High, Katherine A., Arruda, Valder R.

“…Emerging data from early phase clinical studies of AAV gene therapy for hemophilia B (HB) (factor IX [FIX] deficiency) show sustained expression of therapeutic…”
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Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression by Crudele, Julie M, Van Sluis, Geerte L., Margaritis, Paris, Siner, Joshua I, Sliozberg, Michael, Maurer, Jenna, Faella, Armida, Zhou, Shangzhen, High, Katherine A., Esmon, Charles T, Spek, C. Arnold, Arruda, Valder R

“…Abstract 3350 Cancer is frequently associated with activation of coagulation, and a procoagulant state facilitates tumor metastasis. Recent studies have…”
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