Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

[1]  J. Oldenburg,et al.  A multicenter, open-label, phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. , 2019, Blood.

[2]  S. Kaveri,et al.  Correction of bleeding in experimental severe hemophilia A by systemic delivery of factor VIII-encoding mRNA , 2019, Haematologica.

[3]  C. Lukacs,et al.  mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency. , 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

[4]  M. Makris,et al.  How to discuss gene therapy for haemophilia? A patient and physician perspective , 2019, Haemophilia : the official journal of the World Federation of Hemophilia.

[5]  K. Chien,et al.  Intradermal delivery of modified mRNA encoding VEGF-A in patients with type 2 diabetes , 2019, Nature Communications.

[6]  Kimberly J. Hassett,et al.  Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines , 2019, Molecular therapy. Nucleic acids.

[7]  Hande Kızılocak,et al.  Management of perioperative hemostasis in a severe hemophilia A patient with inhibitors on emicizumab using global hemostasis assays , 2019, Therapeutic advances in hematology.

[8]  L. Aledort,et al.  Evaluating the safety of emicizumab in patients with hemophilia A , 2018, Expert opinion on drug safety.

[9]  Yuan-Yang Cheng,et al.  Outcome of perioperative hemostatic management in patients with hemophilia without inhibitors undergoing 161 invasive or surgical procedures , 2018, Journal of the Chinese Medical Association.

[10]  C. Casari,et al.  A factor VIII-nanobody fusion protein forming an ultrastable complex with VWF: effect on clearance and antibody formation. , 2018, Blood.

[11]  Anton P. McCaffrey,et al.  Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification , 2018, Molecular therapy. Nucleic acids.

[12]  P. Neumann,et al.  Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia , 2018, Journal of managed care & specialty pharmacy.

[13]  V. Jiménez‐Yuste,et al.  Recommendations on multidisciplinary management of elective surgery in people with haemophilia , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[14]  P. Cullis,et al.  Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility. , 2018, Nucleic acid therapeutics.

[15]  Örn Almarsson,et al.  A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates. , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[16]  E. James,et al.  FVIII proteins with a modified immunodominant T-cell epitope exhibit reduced immunogenicity and normal FVIII activity. , 2018, Blood advances.

[17]  H. Lee,et al.  Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine. , 2018, Biomaterials.

[18]  G. Ronzitti,et al.  Emerging Issues in AAV-Mediated In Vivo Gene Therapy , 2017, Molecular therapy. Methods & clinical development.

[19]  A. Schambach,et al.  Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy , 2017, Molecular therapy. Methods & clinical development.

[20]  C. Lukacs,et al.  Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia , 2017, Cell reports.

[21]  L. George,et al.  Hemophilia gene therapy comes of age. , 2017, Blood advances.

[22]  D. Perry,et al.  AAV5–Factor VIII Gene Transfer in Severe Hemophilia A , 2017, The New England journal of medicine.

[23]  C. Rothschild,et al.  Analyses of the FranceCoag cohort support differences in immunogenicity among one plasma-derived and two recombinant factor VIII brands in boys with severe hemophilia A , 2017, Haematologica.

[24]  C. Kempton,et al.  Current and emerging factor VIII replacement products for hemophilia A , 2017, Therapeutic advances in hematology.

[25]  J. Tarrant,et al.  Biological considerations of plasma-derived and recombinant factor VIII immunogenicity. , 2017, Blood.

[26]  K. Lieuw.  Many factor VIII products available in the treatment of hemophilia A: an embarrassment of riches? , 2017, Journal of blood medicine.

[27]  E. Santagostino,et al.  Outcome of Clinical Trials with New Extended Half-Life FVIII/IX Concentrates , 2017, Journal of clinical medicine.

[28]  I. Verma,et al.  Systemic delivery of factor IX messenger RNA for protein replacement therapy , 2017, Proceedings of the National Academy of Sciences.

[29]  V. Arruda,et al.  Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A , 2016, Journal of thrombosis and haemostasis : JTH.

[30]  H. Wada,et al.  Emicizumab Prophylaxis in Hemophilia A with Inhibitors. , 2017, The New England journal of medicine.

[31]  Kevin J. Kauffman,et al.  Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system , 2016, Gene Therapy.

[32]  D. Covas,et al.  The F309S mutation increases factor VIII secretion in human cell line , 2016, Revista brasileira de hematologia e hemoterapia.

[33]  D. Rawlings,et al.  Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A. , 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

[34]  A. Elkahloun,et al.  Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. , 2015, The Journal of clinical investigation.

[35]  V. Mauro,et al.  A critical analysis of codon optimization in human therapeutics. , 2014, Trends in molecular medicine.

[36]  Özlem Türeci,et al.  mRNA-based therapeutics — developing a new class of drugs , 2014, Nature Reviews Drug Discovery.

[37]  C. Miao,et al.  Long‐term tolerance to factor VIII is achieved by administration of interleukin‐2/interleukin‐2 monoclonal antibody complexes and low dosages of factor VIII , 2014, Journal of thrombosis and haemostasis : JTH.

[38]  Y. Jeng,et al.  Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo , 2013, Thrombosis and Haemostasis.

[39]  K. L. La Perle,et al.  Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[40]  O. Rawley,et al.  Elevated factor VIII levels and risk of venous thrombosis , 2012, British journal of haematology.

[41]  A. Bitonti,et al.  Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. , 2012, Blood.

[42]  J. Dumont,et al.  Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. , 2012, Blood.

[43]  A. Thrasher,et al.  Codon optimization of human factor VIII cDNAs leads to high-level expression. , 2011, Blood.

[44]  H. Kazazian,et al.  Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model. , 2009, Blood.

[45]  Hiroki Kato,et al.  Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

[46]  G. Freeman,et al.  Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice. , 2008, Blood.

[47]  Daniel G. Miller,et al.  AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma , 2007, Science.

[48]  A. Judge,et al.  Hypersensitivity and loss of disease site targeting caused by antibody responses to PEGylated liposomes. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

[49]  J. Heyes,et al.  Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. , 2005, Biochimica et biophysica acta.

[50]  S. Pipe,et al.  1 Bioengineering of Coagulation Factor VIII for Improved Secretion , 2004, Pediatric Research.

[51]  H. Ochs,et al.  Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[52]  R. Kaufman,et al.  ATP-dependent dissociation of non-disulfide-linked aggregates of coagulation factor VIII is a rate-limiting step for secretion. , 2000, Biochemistry.

[53]  E. Tuddenham.  Molecular Biological Aspects of Inhibitor Development , 1999, Vox Sanguinis.

[54]  A. Berns,et al.  Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. , 1995, Proceedings of the National Academy of Sciences of the United States of America.

[55]  G. Auerswald,et al.  Factor VIII Inhibitors in Patients with Hemophilia A: Epidemiology of Inhibitor Development and Induction of Immune Tolerance for Factor VIII , 1995, Seminars in thrombosis and hemostasis.

[56]  A. Miller,et al.  Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. , 1993, Human gene therapy.

[57]  W. Kreuz,et al.  Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs , 1992, The Lancet.

[58]  K Fujikawa,et al.  The coagulation cascade: initiation, maintenance, and regulation. , 1991, Biochemistry.

[59]  A. Dorner,et al.  Increased synthesis of secreted proteins induces expression of glucose-regulated proteins in butyrate-treated Chinese hamster ovary cells. , 1989, The Journal of biological chemistry.

[60]  A. Dorner,et al.  Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. , 1988, The Journal of biological chemistry.