Effect of Fviii Co-administrated with Ivig in Immunity to Fviii in Hemophilia a Mice Effect of Fviii Co-administrated with Ivig in Immunity to Fviii in Hemophilia a Mice
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چکیده
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منابع مشابه
Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.
Immune responses to coagulation factors VIII (FVIII) and IX (FIX) represent primary obstacles to hemophilia treatment. Previously, we showed that hematopoietic stem cell (HSC) retroviral gene therapy induces immune nonresponsiveness to FVIII in both naive and preimmunized murine hemophilia A settings. Liver-directed adeno-associated viral (AAV)-FIX vector gene transfer achieved similar results ...
متن کاملTolerogenic nanoparticles to induce immunologic tolerance: Prevention and reversal of FVIII inhibitor formation.
The immune response of hemophilia A patients to administered FVIII is a major complication that obviates this very therapy. We have recently described the use of synthetic, biodegradable nanoparticles carrying rapamycin and FVIII peptide antigens, to induce antigen-specific tolerance. Herein we test the tolerogenicity of nanoparticles that contains full length FVIII protein in hemophilia A mice...
متن کاملA prospective Crossover Triple-blind Controlled Trial on the Safety and Efficacy of Iranian Recombinant FVIII (Safacto) versus Plasma Derived FVIII A pilot study
Background: Considering the increasing number of patients with hemophilia and infrastructure requirements for a comprehensive approach, development of a recombinant factor has become a milestone. The objective of this study was to assess the safety, efficacy and non inferiority of Safacto (Recombinant factor VIII) compared with plasma-derived factor in the treatment of hemophilia A. Methods: 10 ...
متن کاملGENE THERAPY Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity
Although genetic induction of factor VIII (FVIII) expression in platelets can restore hemostasis in hemophilia A mice, this approach has not been studied in the clinical setting of preexisting FVIII inhibitory antibodies to determine whether such antibodies would affect therapeutic engraftment. We generated a line of transgenic mice (2bF8) that express FVIII only in platelets using the platelet...
متن کاملBioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery
Clinical data support the feasibility and safety of adeno-associated viral (AAV) vectors in gene therapy applications. Despite several clinical trials of AAV-based gene transfer for hemophilia B, a unique set of obstacles impede the development of a similar approach for hemophilia A. These include (i) the size of the factor VIII (fVIII) transgene, (ii) humoral immune responses to fVIII, (iii) i...
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تاریخ انتشار 2016