Induction of tolerance to factor VIII by transient co‐administration with rapamycin

See also Miao CH. Tilt balance towards regulation: evolving new strategy for treatment of hemophilia inhibitors. This issue, pp 1521–3.DOI:10.1111/j.1538‐7836.2011.04351.x.

[1]  D. Scott,et al.  B-cell-delivered gene therapy induces functional T regulatory cells and leads to a loss of antigen-specific effector cells. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

[2]  K. Peerlinck,et al.  Mild haemophilia: a disease with many faces and many unexpected pitfalls , 2010, Haemophilia : the official journal of the World Federation of Hemophilia.

[3]  S. Kaveri,et al.  Induction of heme oxygenase-1 in factor VIII-deficient mice reduces the immune response to therapeutic factor VIII. , 2010, Blood.

[4]  H. Daniell,et al.  Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice , 2010, Proceedings of the National Academy of Sciences.

[5]  M. Franchini,et al.  Understanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategies , 2010, Haemophilia : the official journal of the World Federation of Hemophilia.

[6]  H. Ochs,et al.  Anti-CD3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy. , 2009, Blood.

[7]  S. Ziegler,et al.  CD4+FOXP3+ regulatory T cells confer long-term regulation of factor VIII-specific immune responses in plasmid-mediated gene therapy-treated hemophilia mice. , 2009, Blood.

[8]  H. Ertl,et al.  Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[9]  M. Atkinson,et al.  Prophylactic immune tolerance induced by changing the ratio of antigen‐specific effector to regulatory T cells , 2009, Journal of thrombosis and haemostasis : JTH.

[10]  E. James,et al.  Lineages of human T-cell clones, including T helper 17/T helper 1 cells, isolated at different stages of anti-factor VIII immune responses. , 2009, Blood.

[11]  B. Brown,et al.  A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells. , 2009, Blood.

[12]  R. Herzog,et al.  Role of regulatory T cells in tolerance to coagulation factors , 2009, Journal of thrombosis and haemostasis : JTH.

[13]  D. Wechsler,et al.  Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome , 2009, British journal of haematology.

[14]  K. Ghosh,et al.  Immune Response to FVIII in Hemophilia A: An Overview of Risk Factors , 2009, Clinical reviews in allergy & immunology.

[15]  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.

[16]  E. James,et al.  T‐cell responses over time in a mild hemophilia A inhibitor subject: epitope identification and transient immunogenicity of the corresponding self‐peptide , 2007, Journal of thrombosis and haemostasis : JTH.

[17]  K. Ponder,et al.  High expression reduces an antibody response after neonatal gene therapy with B domain‐deleted human factor VIII in mice , 2007, Journal of thrombosis and haemostasis : JTH.

[18]  R. Herzog,et al.  Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. , 2007, Blood.

[19]  M. Battaglia,et al.  Rapamycin Promotes Expansion of Functional CD4+CD25+FOXP3+ Regulatory T Cells of Both Healthy Subjects and Type 1 Diabetic Patients1 , 2006, The Journal of Immunology.

[20]  D. DiMichele.  Immune tolerance: critical issues of factor dose, purity and treatment complications , 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

[21]  K. Sullivan,et al.  Rapamycin improves lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome (ALPS). , 2006, Blood.

[22]  H. Ochs,et al.  Immunomodulation of transgene responses following naked DNA transfer of human factor VIII into hemophilia A mice. , 2005, Blood.

[23]  D. Scott,et al.  Induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins. , 2005, Blood.

[24]  Jun Liu,et al.  Rapamycin Induces Heme Oxygenase-1 in Human Pulmonary Vascular Cells: Implications in the Antiproliferative Response to Rapamycin , 2003, Circulation.

[25]  A. Sharpe,et al.  Prevention and treatment of factor VIII inhibitors in murine hemophilia AJ. Qian, M. Collins, A.H. Sharpe, et al. Blood 95:1324–1329, 2000 , 2000 .

[26]  H. Kazazian,et al.  Inhibitor Antibody Development and T Cell Response to Human Factor VIII in Murine Hemophilia A , 1999, Thrombosis and Haemostasis.

[27]  S. Antonarakis,et al.  Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A , 1995, Nature Genetics.

[28]  P. Thompson,et al.  Anti-CD3 prevents factor VIII inhibitor development in hemophilia A mice by a regulatory CD4+CD25+-dependent mechanism and by shifting cytokine production to favor a Th1 response. , 2009, Blood.

[29]  R. Negrin,et al.  Differential impact of mammalian target of rapamycin inhibition on CD4+CD25+Foxp3+ regulatory T cells compared with conventional CD4+ T cells. , 2008, Blood.