Search Results - "Iacobelli, Nicholas P."

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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X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua) by Simioni, Paolo, Tormene, Daniela, Tognin, Giulio, Gavasso, Sabrina, Bulato, Cristiana, Iacobelli, Nicholas P, Finn, Jonathan D, Spiezia, Luca, Radu, Claudia, Arruda, Valder R

“…A 23-year-old man with deep-vein thrombosis was found to have a gain-of-function mutation in his factor IX gene. The plasma level of factor IX was normal, but…”
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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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Understanding Ectopically Expressed Factor VIII (F8) In Megakaryocytes: Implications for Optimum Platelet-Delivered F8 Activity for Gene Therapy by Greene, Teshell K, Sabatino, Denise E., Iacobelli, Nicholas P, Zhai, Li, Pipe, Steven W., Kowalska, M. Anna, Camire, Rodney M., Arruda, Valder

“…Abstract 2205 F8 ectopically expressed during megakaryopoiesis is stored in platelet (p) a-granules and released at sites of injury. This F8 is effective in…”
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X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)Brief Report by Simioni, Paolo, Tormene, Daniela, Tognin, Giulio, Gavasso, Sabrina, Bulato, Cristiana, Iacobelli, Nicholas P, Finn, Jonathan D, Spiezia, Luca, Radu, Claudia, Arruda, Valder R

“…We report a case of juvenile thrombophilia associated with a substitution of leucine for arginine at position 338 (R338L) in the factor IX gene (factor…”
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