Gene therapy for immune tolerance induction in hemophilia with inhibitors

In vivo induction of regulatory T cells for immune tolerance in hemophilia

(2016) Xiaomei Wang et al.   CELLULAR IMMUNOLOGY

Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies

(2016) T. C. Nichols et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Impact of inhibitors on hemophilia a mortality in the United States

(2015) Christopher E. Walsh et al.   AMERICAN JOURNAL OF HEMATOLOGY

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

(2015) J. M. Crudele et al.   BLOOD

FVIII inhibitors: pathogenesis and avoidance

(2015) J. Astermark   BLOOD

Inhibitor development and mortality in non-severe hemophilia A

(2015) C. L. Eckhardt et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Inhibitor recurrence after immune tolerance induction: a multicenter retrospective cohort study

(2015) A. Antun et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A

(2015) Xuefeng Wang et al.   MOLECULAR THERAPY

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

(2015) Alessio Cantore et al.   Science Translational Medicine

Hurdles in therapy with regulatory T cells

(2015) Piotr Trzonkowski et al.   Science Translational Medicine

Obstacles and future of gene therapy for hemophilia

(2015) Valder R Arruda et al.   Expert Opinion on Orphan Drugs

A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII

(2014) S. A. Fahs et al.   BLOOD

Murine coagulation factor VIII is synthesized in endothelial cells

(2014) L. A. Everett et al.   BLOOD

Affinity of FVIII-specific antibodies reveals major differences between neutralizing and nonneutralizing antibodies in humans

(2014) C. J. Hofbauer et al.   BLOOD

Toward optimal therapy for inhibitors in hemophilia

(2014) C. L. Kempton et al.   BLOOD

Engineered antigen-specific human regulatory T cells: immunosuppression of FVIII-specific T- and B-cell responses

(2014) Y. C. Kim et al.   BLOOD

Immune Tolerance Induction to Factor IX through B Cell Gene Transfer: TLR9 Signaling Delineates between Tolerogenic and Immunogenic B Cells

(2014) Xiaomei Wang et al.   MOLECULAR THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Development of gene transfer for induction of antigen-specific tolerance

(2014) Brandon K Sack et al.   Molecular Therapy-Methods & Clinical Development

Progress toward inducing immunologic tolerance to factor VIII

(2013) D. W. Scott et al.   BLOOD

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

(2013) J. McIntosh et al.   BLOOD

Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A

(2013) C. L. Eckhardt et al.   BLOOD

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

(2013) David M. Markusic et al.   EMBO Molecular Medicine

Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

(2013) Andrea Annoni et al.   EMBO Molecular Medicine

Platelet Gene Therapy by Lentiviral Gene Delivery to Hematopoietic Stem Cells Restores Hemostasis and Induces Humoral Immune Tolerance in FIXnull Mice

(2013) Yingyu Chen et al.   MOLECULAR THERAPY

Endogenous factor VIII synthesis from the intron 22–inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A

(2013) Gouri Shankar Pandey et al.   NATURE MEDICINE

Increased burden on caregivers of having a child with haemophilia complicated by inhibitors

(2013) Karin Lindvall et al.   PEDIATRIC BLOOD & CANCER

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

(2013) Lily M. Du et al.   Nature Communications

Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients

(2012) S. F. J. Whelan et al.   BLOOD

F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis

(2012) S. C. Gouw et al.   BLOOD

Immune tolerance in haemophilia: the long journey to the fork in the road

(2012) Donna M. DiMichele   BRITISH JOURNAL OF HAEMATOLOGY

Models for assessing immunogenicity and efficacy of new therapeutics for the treatment of haemophilia

(2012) JEAN-MARIE SAINT-REMY et al.   HAEMOPHILIA

Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity

(2012) E. L. KUETHER et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

(2012) Brandon K. Sack et al.   PLoS One

Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United Kingdom

(2011) C. R. M. Hay et al.   BLOOD

The principal results of the International Immune Tolerance Study: a randomized dose comparison

(2011) C. R. M. Hay et al.   BLOOD

Healthcare expenditures for males with haemophilia and employer-sponsored insurance in the United States, 2008

(2011) S. GUH et al.   HAEMOPHILIA

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Factor IX ectopically expressed in platelets can be stored in  -granules and corrects the phenotype of hemophilia B mice

(2010) G. Zhang et al.   BLOOD

In vivo efficacy of platelet-delivered, high specific activity factor VIII variants

(2010) T. K. Greene et al.   BLOOD

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

(2010) J. D. Finn et al.   BLOOD

Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States

(2010) Char Witmer et al.   BRITISH JOURNAL OF HAEMATOLOGY

Factor VIII-specific B cell responses in haemophilia A patients with inhibitors

(2010) P. M. W. Van HELDEN et al.   HAEMOPHILIA

Animal models of inhibitors

(2010) B. REIPERT et al.   HAEMOPHILIA

Immunoglobulin isotypes and functional anti-FVIII antibodies in response to FVIII treatment in Balb/c and C57BL/6 haemophilia A mice

(2010) M. QADURA et al.   HAEMOPHILIA

Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report

(2010) J. ASTERMARK et al.   HAEMOPHILIA

Functional aspects of factor VIII expression after transplantation of genetically-modified hematopoietic stem cells for hemophilia A

(2010) Lucienne M. Ide et al.   JOURNAL OF GENE MEDICINE

Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

(2010) Denise E Sabatino et al.   MOLECULAR THERAPY

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

(2009) A. Annoni et al.   BLOOD

The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy

(2009) D. DIMICHELE   HAEMOPHILIA

Haemopoietic stem cell transplantation induces tolerance to donor antigens but not to foreign FVIII peptides

(2009) J. UPRICHARD et al.   HAEMOPHILIA

Immune tolerance: What is unique about the liver

(2009) Gisa Tiegs et al.   JOURNAL OF AUTOIMMUNITY

Factor VIII gene (F8) mutations as predictors of outcome in immune tolerance induction of hemophilia A patients with high-responding inhibitors

(2009) A. COPPOLA et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

The molecular mechanisms of immunomodulation and tolerance induction to factor VIII

(2009) B. WATERS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

(2009) Paolo Simioni et al.   NEW ENGLAND JOURNAL OF MEDICINE

Dynamics of factor VIII interactions determine its immunologic fate in hemophilia A

(2008) S. Lacroix-Desmazes et al.   BLOOD

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

(2008) G. P. Niemeyer et al.   BLOOD

Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors

(2008) J. GEWIRTZ et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Arthropathy in Inhibitor Patients: Differences in the Joint Status

(2008) W. Keith Hoots   SEMINARS IN HEMATOLOGY