Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A

SW Pipe, RR Montgomery, KP Pratt… - Blood, The Journal …, 2016 - ashpublications.org
A normal hemostatic response to vascular injury requires both factor VIII (FVIII) and von
Willebrand factor (VWF). In plasma, VWF and FVIII normally circulate as a noncovalent …

Tolerating factor VIII: recent progress

S Lacroix-Desmazes, J Voorberg, D Lillicrap… - Frontiers in …, 2020 - frontiersin.org
Development of neutralizing antibodies against biotherapeutic agents administered to
prevent or treat various clinical conditions is a longstanding and growing problem faced by …

Chitosan derivative composite nanoparticles as adjuvants enhance the cellular immune response via activation of the cGAS-STING pathway

Z Zhao, Y Peng, X Shi, K Zhao - International Journal of Pharmaceutics, 2023 - Elsevier
Chitosan and its derivatives are widely used in vaccine adjuvants and delivery systems.
Vaccine antigens encapsulated in or conjugated onto N-2-hydroxypropyl trimethyl …

Factor VIII trafficking to CD4+ T cells shapes its immunogenicity and requires several types of antigen-presenting cells

R Kaczmarek, AR Piñeros, PE Patterson… - Blood, The Journal …, 2023 - ashpublications.org
Despite> 80 years of clinical experience with coagulation factor VIII (FVIII) inhibitors,
surprisingly little is known about the in vivo mechanism of this most serious complication of …

Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice

AR Sternberg, C Martos-Rus, RJ Davidson… - Nature …, 2024 - nature.com
Durable factor VIII expression that normalizes hemostasis is an unrealized goal of
hemophilia A adeno-associated virus-mediated gene therapy. Trials with initially normal …

Marginal zone B cells are critical to factor VIII inhibitor formation in mice with hemophilia A

PE Zerra, C Cox, WH Baldwin, SR Patel… - Blood, The Journal …, 2017 - ashpublications.org
Although factor VIII (FVIII) replacement therapy can be lifesaving for patients with hemophilia
A, neutralizing alloantibodies to FVIII, known as inhibitors, develop in a significant number of …

[HTML][HTML] On the versatility of von Willebrand factor

A Rauch, N Wohner, OD Christophe… - … journal of hematology …, 2013 - ncbi.nlm.nih.gov
Von Willebrand factor (VWF) is a large multimeric protein, the function of which has been
demonstrated to be pivotal to the haemostatic system. Indeed, quantitative and/or qualitative …

Biological considerations of plasma-derived and recombinant factor VIII immunogenicity

J Lai, C Hough, J Tarrant… - Blood, The Journal of the …, 2017 - ashpublications.org
In hemophilia A, the most severe complication of factor VIII (FVIII) replacement therapy
involves the formation of FVIII neutralizing antibodies, also known as inhibitors, in 25% to …

Marginal zone B cells induce alloantibody formation following RBC transfusion

SR Patel, DR Gibb, K Girard-Pierce, X Zhou… - Frontiers in …, 2018 - frontiersin.org
Red blood cell (RBC) alloimmunization represents a significant immunological challenge for
some patients. While a variety of immune constituents likely contribute to the initiation and …

High-affinity, noninhibitory pathogenic C1 domain antibodies are present in patients with hemophilia A and inhibitors

G Batsuli, W Deng, JF Healey, ET Parker… - Blood, The Journal …, 2016 - ashpublications.org
Inhibitor formation in hemophilia A is the most feared treatment-related complication of factor
VIII (fVIII) therapy. Most inhibitor patients with hemophilia A develop antibodies against the …