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Showing 1 to 16 of 16 for “"Hemophilia A"”.
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SUBLINGUAL IMMUNOTHERAPY INDUCES TOLERANCE TO FACTOR VIII IN HEMOPHILIA A MICE
Hemophilia A (HA) is a rare X-linked recessive genetic bleeding disorder characterized by the absence or decreased amount of functional coagulation Factor VIII (FVIII) in the blood. The clinical manifestations of HA include uncontrolled bleeding events, spontaneous muscle bleeding, and joint …
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Improving the Safety and Efficacy of AAV Gene Therapy for Hemophilia A
Gene therapy for hemophilia A (HA) remains an unrealized therapeutic goal due to outstanding questions regarding safety and long-term efficacy after adeno-associated viral (AAV) vector delivery of the F8 gene to hepatocytes. To address questions of safety, we treated privately owned dogs with a …
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PIECING TOGETHER THE PUZZLE OF TREATING HEMOPHILIA A UTILIZING AAV BASED GENE THERAPY
… mutation or deletion. Disorders range from Hemophilia, Cystic fibrosis, and Duchenne muscular dystrophy to diseases like Cancer and Huntington’s Disease. While these diseases may seem vastly different, they all are due to issues in the genetic makeup of the patient. Gene therapy treats or …
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ENHANCEMENT OF hFVIII ACTIVITY THROUGH LC MODIFICATIONS FOR GENE THERAPY OF HEMOPHILIA A
Gene therapy for Hemophilia A (HA) using the recombinant Adeno-associated virus (rAAV) offers an alternative 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 …
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Evaluation of the determinants and characterization of bleeding in hemophilia A and B carriers
Hemophilia A and B are bleeding disorders caused 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 …
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Stimulation Through Tlr4 Increases Fviii Inhibitor Formation In A Mouse Model of Hemophilia A
<p>Hemophilia A is a clotting 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 …
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Infusing Factor Viii-Expressing Platelets Or Megakaryocytes As A Novel Therapeutic Strategy For Hemophilia A
… most common inherited form of severe bleeding, hemophilia A, a deficiency of functional coagulation factor VIII. Patients with severe hemophilia A suffer from recurrent bleeding with significant morbidity and mortality with 20-30% of these patients developing antibodies to infused Factor (F) …
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GLOBAL COAGULATION ASSAYS AND HEMOPHILIA ARTHROPATHY SCORES FOR MONITORING EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH HEMOPHILIA A
Introduzione: Le terapie non sostitutive stanno emergendo come valido trattamento nei pazienti affetti da emofilia. Tuttavia, nonostante i risultati promettenti, non esiste una tecnica di monitoraggio. La prima terapia non sostitutiva approvata per il trattamento dei pazienti affetti da emofilia A …
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Characterization of the Recombinant Human Factor VIII Expressed in the Milk of Transgenic Swine
… therapeutic applications for the treatment of Hemophilia A. Its deficiency, either qualitative or quantitative, results in Hemophilia A, a disorder affecting approximately 1 in 10,000 males. Currently, FVIII replacement therapy uses FVIII derived from plasma or cell culture. The current cost of …
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FVIII Immunity : early events and tolerance mechanisms to FVIII
Among the complications of current treatments 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 …
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A platelet-mediated paradigm for the targeted delivery of microencapsulated, clot-augmenting biotherapeutics
… 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 it is …
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Computational Modeling And Simulation Of Thrombus Formation
… patient-derived plasma for coronary artery and hemophilia A patients. In Chapter 5, analysis of the sensitivity results discloses that the intrinsic protease factor XI could be an excellent therapeutic target for thrombosis treatment with the advantage of not affecting hemostasis. Chapter 6 …
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Phosphatidylserine-Based Nanoparticles For Tolerance Induction Towards Therapeutic Proteins
… dysfunction or deficiency of FVIII manifests in Hemophilia A (HA). Despite enzyme replacement therapy as the first line of therapy, about 30% of severe HA patients develop neutralizing (NAbs) or inhibitory titers, severely complicating efficacy of therapy. As a result, any approach designed to …
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In Vivo Genome Editing: Proof of Concept in Neonatal and Adult Mouse Liver
… gene delivery for monogenic diseases, including hemophilia. Major limitations of this approach are the inability to persist in dividing cells and the restrictive packaging capacity of AAV. Gene targeting, the ability to make site-directed changes to the genome, has been a powerful tool for …
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Exploring Anti-FVIII Antibodies in Haemophilia A - Role in In Vitro Haemostasis and Clinical Disease
Haemophilia A (HA) is caused by 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 …
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COMPUTATIONAL APPROACHES IN STUDYING INHIBITOR DEVELOPMENT IN HAEMOPHILIA A PATIENTS
L'emofilia A è un disturbo emorragico recessivo legato al cromosoma X, causato da mutazioni nel gene F8, che determinano una carenza o un malfunzionamento del fattore VIII (FVIII) della coagulazione. Una delle principali complicazioni cliniche nei casi gravi è lo sviluppo di alloanticorpi …