Laboratory testing for factor VIII and IX inhibitors in haemophilia: a review

CH Miller - Haemophilia, 2018 - Wiley Online Library
Inhibitors are antibodies directed against haemophilia treatment products which interfere
with their function. Factor VIII (FVIII) inhibitors in haemophilia A and factor IX (FIX) inhibitors …

Plasma clearance of coagulation factor VIII and extension of its half-life for the therapy of hemophilia A: A critical review of the current state of research and practice

AG Sarafanov - International Journal of Molecular Sciences, 2023 - mdpi.com
Factor VIII (FVIII) is an important component of blood coagulation as its congenital deficiency
results in life-threatening bleeding. Current prophylactic therapy of the disease (hemophilia …

Codon optimization of human factor VIII cDNAs leads to high-level expression

NJ Ward, SMK Buckley, SN Waddington… - Blood, The Journal …, 2011 - ashpublications.org
Gene therapy for hemophilia A would be facilitated by development of smaller expression
cassettes encoding factor VIII (FVIII), which demonstrate improved biosynthesis and/or …

Characterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIII

C Kannicht, M Ramström, G Kohla, M Tiemeyer… - Thrombosis research, 2013 - Elsevier
INTRODUCTION: Host cell lines used for recombinant protein expression differ in their
ability to perform post-translational modifications (PTMs). The currently available …

Suppression of inhibitor formation against FVIII in a murine model of hemophilia A by oral delivery of antigens bioencapsulated in plant cells

A Sherman, J Su, S Lin, X Wang… - Blood, The Journal …, 2014 - ashpublications.org
Hemophilia A is the X-linked bleeding disorder caused by deficiency of coagulation factor
VIII (FVIII). To address serious complications of inhibitory antibody formation in current …

The first recombinant human coagulation factor VIII of human origin: human cell line and manufacturing characteristics

E Casademunt, K Martinelle, M Jernberg… - European journal of …, 2012 - Wiley Online Library
Introduction Since the early 1990s, recombinant human clotting factor VIII (rhFVIII) produced
in hamster cells has been available for haemophilia A treatment. However, the post …

[HTML][HTML] Factor VIII and von Willebrand factor are ligands for the carbohydrate-receptor Siglec-5

JN Pegon, M Kurdi, C Casari, S Odouard… - …, 2012 - ncbi.nlm.nih.gov
Background Factor VIII (FVIII) and von Willebrand factor (VWF) circulate in plasma in a tight
non-covalent complex, being critical to hemostasis. Although structurally unrelated, both …

[HTML][HTML] Plant cell-made protein antigens for induction of Oral tolerance

H Daniell, M Kulis, RW Herzog - Biotechnology advances, 2019 - Elsevier
The gut associated lymphoid tissue has effective mechanisms in place to maintain tolerance
to food antigens. These can be exploited to induce antigen-specific tolerance for the …

Protein-engineered coagulation factors for hemophilia gene therapy

BJ Samelson-Jones, VR Arruda - Molecular Therapy-Methods & Clinical …, 2019 - cell.com
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to
inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively …

How to translate and implement the current science of gene therapy into haemophilia care?

C Hermans, Y Gruel, L Frenzel… - Therapeutic advances …, 2023 - journals.sagepub.com
Gene-based therapy opens an entirely new paradigm in managing people with haemophilia
(PWH), offering them the possibility of a functional cure by enabling continuous expression …