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 …

Advances in knowledge of inhibitor formation in severe haemophilia A

M Cormier, P Batty, J Tarrant… - British Journal of …, 2020 - Wiley Online Library
Anti‐drug antibody formation following factor VIII (FVIII) replacement therapy is the most
important treatment‐related complication in patients with severe haemophilia A. A significant …

The endothelial cell receptor stabilin-2 regulates VWF-FVIII complex half-life and immunogenicity

LL Swystun, JD Lai, C Notley… - The Journal of …, 2018 - Am Soc Clin Investig
Quantitative abnormalities of the von Willebrand factor–factor VIII (VWF-FVIII) complex
associate with inherited bleeding or thrombotic disorders. Receptor-mediated interactions …

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 …

Timing of intensive immunosuppression impacts risk of transgene antibodies after AAV gene therapy in nonhuman primates

BJ Samelson-Jones, JD Finn, P Favaro… - … Therapy Methods & …, 2020 - cell.com
Adeno-associated virus (AAV) vector gene therapy is a promising treatment for a variety of
genetic diseases, including hemophilia. Systemic administration of AAV vectors is …

[HTML][HTML] The endothelial lectin clearance receptor CLEC4M binds and internalizes factor VIII in a VWF‐dependent and independent manner

LL Swystun, C Notley, I Georgescu, JD Lai… - Journal of Thrombosis …, 2019 - Elsevier
Essentials• CLEC4M is an endocytic receptor for factor FVIII.• CLEC4M interacts with FVIII in
a VWF‐dependent and independent manner.• CLEC4M binds to mannose‐containing …

[HTML][HTML] The contribution of the sinusoidal endothelial cell receptors CLEC4M, stabilin-2, and SCARA5 to VWF–FVIII clearance in thrombosis and hemostasis

LL Swystun, A Michels, D Lillicrap - Journal of Thrombosis and …, 2023 - Elsevier
Quantitative abnormalities in factor VIII (FVIII) and its binding partner, von Willebrand factor
(VWF), are associated with an increased risk of bleeding or thrombosis, and pathways that …

FVIII at the crossroad of coagulation, bone and immune biology: Emerging evidence of biological activities beyond hemostasis

M Cadé, J Munoz-Garcia, A Babuty, M Fouassier… - Drug Discovery …, 2022 - Elsevier
Hemophilia A is an X-linked hereditary disorder that results from deficient coagulation factor
VIII (FVIII) activity, leading to spontaneous bleeding episodes, particularly in joints and …

Novel combinatorial microRNA-binding sites in AAV vectors synergistically diminish antigen presentation and transgene immunity for efficient and stable transduction

M Muhuri, W Zhan, Y Maeda, J Li, A Lotun… - Frontiers in …, 2021 - frontiersin.org
Recombinant adeno-associated virus (rAAV) platforms hold promise for in vivo gene therapy
but are undermined by the undesirable transduction of antigen presenting cells (APCs) …

Induction of activated T follicular helper cells is critical for anti-FVIII inhibitor development in hemophilia A mice

W Jing, J Chen, Y Cai, Y Chen, JA Schroeder… - Blood …, 2019 - ashpublications.org
The development of neutralizing anti-FVIII antibodies (inhibitors) is a major complication of
FVIII protein replacement therapy in patients with hemophilia A (HA). Although multiple lines …