Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
Results
Showing 1 to 20 of 20 for “"FVIII"”.
-
FVIII Immunity : early events and tolerance mechanisms to FVIII
… for hemophilia A, the development of anti-FVIII antibodies including “FVIII inhibitors” remains the major clinical problem in treating hemophiliacs. Factor VIII inhibitors work through neutralizing the coagulation cofactor activity of the infused FVIII and preventing the restoration of …
-
Stimulation Through Tlr4 Increases Fviii Inhibitor Formation In A Mouse Model of Hemophilia A
… disorder caused by functional factor VIII (FVIII) deficiency. About 25% of patients treated with therapeutic recombinant FVIII develop antibodies (inhibitors) that render subsequent FVIII treatments ineffective. The immune mechanisms of inhibitor formation are not entirely understood, but …
-
Exploring Anti-FVIII Antibodies in Haemophilia A - Role in In Vitro Haemostasis and Clinical Disease
… defective synthesis of coagulation factor VIII(FVIII), which has serious effects on haemostasis; joints being the most common site of bleeding. The development of FVIII replacements has improved the situation for patients with haemophilia such that chronic arthropathy can be prevented, and life …
-
Characterization of the Recombinant Human Factor VIII Expressed in the Milk of Transgenic Swine
… approximately 1 in 10,000 males. Currently, FVIII replacement therapy uses FVIII derived from plasma or cell culture. The current cost of this therapy is in excess of $150,000 per patient per year. Thus, alternative sources that are more economical are attractive. The present work focuses …
-
Improving the Safety and Efficacy of AAV Gene Therapy for Hemophilia A
… that selective pressures keep factor VIII (FVIII) activity low. Using criteria established from studies of factor IX-Padua, a factor IX transgene with increased specific activity, we identified 52 amino acids we hypothesized would lead to increased FVIII activity when substituted. A screen …
-
A platelet-mediated paradigm for the targeted delivery of microencapsulated, clot-augmenting biotherapeutics
… the pro-clotting biotherapeutic, factor VIII (fVIII) for hemophilia A patients who have developed inhibitory anti-fVIII antibodies. The polyelectrolyte multilayer capsules physically shield the encapsulated fVIII from the patient’s inhibitors during circulation, preserving its bioactivity until …
-
ENHANCEMENT OF hFVIII ACTIVITY THROUGH LC MODIFICATIONS FOR GENE THERAPY OF HEMOPHILIA A
… to classic treatment, which consists of FVIII protein infusions. However, due to limitations associated with rAAV and the FVIII protein itself, the end result is a transgene expression below therapeutic limits. One approach to improving the therapeutic value of rAAV gene therapy for HA is …
-
Θρομβοελαστογραφία ROTEM ως πιθανός δείκτης ποιότητας φρέσκου κατεψυγμένου πλάσματος FFP
Ο παράγοντας VIII (FVIII) αποτελεί τον καθιερωμένο δείκτη ποιότητας για το φρέσκο κατεψυγμένο πλάσμα (FFP). Ωστόσο, η αξιολόγηση της συνολικής αιμοστατικής ικανότητας του πλάσματος περιορίζεται μετρώντας μόνο τον παράγοντα VIII (FVIII). Στόχος της εργασίας ήταν η ολοκληρωμένη εκτίμηση της …
-
COMPUTATIONAL APPROACHES IN STUDYING INHIBITOR DEVELOPMENT IN HAEMOPHILIA A PATIENTS
… carenza o un malfunzionamento del fattore VIII (FVIII) della coagulazione. Una delle principali complicazioni cliniche nei casi gravi è lo sviluppo di alloanticorpi neutralizzanti, noti come inibitori, che compromettono l'efficacia del trattamento e aumentano la morbidità. Questa tesi di …
-
Die Wirkung von niedrig dosiertem Desmopressin auf die durch Acetylsalicylsäure verlängerte Blutungszeit
… (DDAVP) auf die Freisetzung von FVIII und den von Willebrand-Faktor (vWF) ist lange bekannt und gut untersucht. Zur Erklärung dieser Beobachtung wird meist die plättchenadhäsive Wirkung des vWF genannt. Obwohl die Bedeutung der vor¬liegenden Daten zur vWF-/FVIII-Freisetzung für …
-
Evaluation of the determinants and characterization of bleeding in hemophilia A and B carriers
… by a deficiency in coagulation factor VIII (FVIII) or coagulation factor IX (FIX), respectively. Due to the X-linked inheritance pattern, males are affected with the disease, while females are almost exclusively carriers of hemophilia. Although carriers were historically assumed to be …
-
Molecular Characterization and Inhibition of Antibodies Elicited Against Galactosyltransferase Knockout Pig Xenografts
… capable of inhibiting clotting factor VIII (FVIII). Therefore, we subsequently identified FVIII as a novel xenoantigen, and provided a preliminary molecular analysis of the interaction between FVIII and the antibodies elicited after xenotransplantation. Additionally, our analyses strongly …
-
Infusing Factor Viii-Expressing Platelets Or Megakaryocytes As A Novel Therapeutic Strategy For Hemophilia A
… for these patients is ectopically expressing FVIII in megakaryocytes and platelets. This FVIII, termed pFVIII, is stored in alpha granules of platelets and is capable of restoring hemostasis in FVIIInull mice, even in the presence of circulating inhibitors. pFVIII has been proposed to be used …
-
Genetic characterization of families with von Willebrand disease
… of type 2A VWD characterized by very low plasma FVIII and VWF levels and the exclusive presence of the dimeric form of VWF in plasma. The findings define a structural element that is indispensable for VWF multimerization. Inherited dominantly, the N1421K mutation causes type 2M VWD characterized …
-
Evaluation of heamostatic potential of emicizumab in patients with heamophilia A using viscoelastic heamostatic assays
… θεραπεία υποκατάστασης με συμπυκνώματα παράγοντα FVIII συνεχίζει να αποτελεί ακρογωνιαίο λίθο της θεραπευτικής αντιμετώπισης της αιμορροφιλίας Α, εμφανίζει σημαντικούς περιορισμούς, όπως είναι η ανάγκη για συχνές ενδοφλέβιες εγχύσεις λόγω του μικρού χρόνου ημίσειας ζωής των συμπυκνωμάτων, η …
-
PATHOPHYSIOLOGY OF VON WILLEBRAND FACTOR IN BLEEDING AND THROMBOSIS
… of damaged vessels and acting as factor VIII (FVIII) carrier. Quantitative or qualitative alteration of VWF protein is responsible for von Willebrand disease (VWD). Conversely, increased VWF levels have been found to be associated to different thrombotic disorders such as arterial thrombosis, …
-
Understanding the Role of von Willebrand Factor (VWF) in Angiogenesis and Angiodysplasia
… injury and stabilizing coagulation factor VIII (FVIII) in circulation. Deficiency or dysfunction of VWF results in von Willebrand disease (VWD), the most common inherited bleeding disorder, as well as acquired von Willebrand syndrome (AVWS) which arises secondary to medical conditions. Beyond its …
-
RARE BLEEDING DISORDERS: INSIGHTS INTO CLINICAL PRESENTATION, LABORATORY PHENOTYPE, AND GENETIC CHARACTERIZATION
… essere la più comune (23%), mentre FII e FV+FVIII le più rare (6%). Tra 257 varianti geniche uniche, l’86% era patogenica e l’11% mai riportata in precedenza; le varianti missenso predominano (57%), soprattutto negli esoni dei domini catalitici (48%). (II) Nel secondo capitolo abbiamo …
-
Aberrant and Alternative Splicing of von Willebrand Factor
… functional platelet, collagen, and Factor VIII (FVIII) binding without co-expression of wildtype (WT) VWF. Alternative splicing is known to occur in approximately 95% of mammalian multi-exon genes; however, no alternative splice variants have been described for VWF thus far. We hypothesized that …
-
GLOBAL COAGULATION ASSAYS AND HEMOPHILIA ARTHROPATHY SCORES FOR MONITORING EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH HEMOPHILIA A
… è stata misurata con il metodo one-stage per FVIII modificato e calibrato. Il NATEM è stato eseguito in sangue intero citrato entro 30 minuti dal prelievo, dopo l'aggiunta di CaCl2 (100mM). I livelli plasmatici del frammento di protrombina F1+2, un marcatore affidabile della generazione di …