Results 41 to 50 of about 16,303 (230)

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

open access: yesNeuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji   +4 more
wiley   +1 more source

Co-expression of Factor VIII with anti-FVIII Camelid antibody ligands: Effect on expression levels of bio-therapeutic FVIII [PDF]

open access: yes, 2015
Production of recombinant FVIII, the protein that is missing or dysfunctional in haemophilia A patients, is highly inefficient compared to other recombinant clotting factors such as FIX.
Tolley, Caroline
core  

Recombinant Factor VIII Fc Inhibits B Cell Activation via Engagement of the FcγRIIB Receptor

open access: yesFrontiers in Immunology, 2020
The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is a major complication of hemophilia A treatment. The sole clinical therapy to restore FVIII tolerance in patients with inhibitors remains immune tolerance induction ...
Maria T. Georgescu   +7 more
doaj   +1 more source

Recombinant porcine factor VIII: Lessons from the past and place in the management of hemophilia A with inhibitors in 2021

open access: yesResearch and Practice in Thrombosis and Haemostasis, 2021
The most serious complication of factor VIII (FVIII) replacement therapy is the occurrence of anti‐FVIII alloantibodies that can strongly reduce or abolish the effect of human FVIII products.
Yesim Dargaud   +1 more
doaj   +1 more source

Engineering a Therapeutic Protein to Enhance the Study of Anti-Drug Immunity

open access: yesBiomedicines, 2022
The development of anti-drug antibodies represents a significant barrier to the utilization of protein-based therapies for a wide variety of diseases.
Patricia E. Zerra   +8 more
doaj   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

open access: yesPrenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada   +2 more
wiley   +1 more source

Complement C3 is a novel modulator of the anti-factor VIII immune response

open access: yesHaematologica, 2018
Development of neutralizing antibodies against therapeutic Factor VIII (FVIII) is the most serious complication of the treatment of hemophilia A. There is growing evidence to show the multifactorial origin of the anti-FVIII immune response, combining ...
Julie Rayes   +10 more
doaj   +1 more source

Determinants of Immune Tolerance Induction Outcomes in 237 Persons With Severe Hemophilia A

open access: yesEuropean Journal of Haematology, EarlyView.
ABSTRACT Immune tolerance induction (ITI) to eradicate an immune response against factor VIII (FVIII) has been used in hemophilia A (HA) with various success rates, costs, and treatment burden. The present study aimed to identify markers predictive of ITI success.
Marcus Fager Ferrari   +5 more
wiley   +1 more source

Role of factor VIII in the prognosis and survival of patients with hepatocellular carcinoma

open access: yesOncologie
Factor VIII (FVIII) has been associated with disease progression and survival in patients with hepatocellular carcinoma (HCC). This study aimed to evaluate the prognostic value of FVIII levels in patients with HCC.
Nguyen Thi Tuyet Mai   +2 more
doaj   +1 more source

CC improve secretion of FVIII-BDD, FVIII-BDD-eGFP and FVIII-FL in vitro.

open access: yes, 2013
Heterologous CHO cells were incubated with CC at different concentrations. FVIII activity was determined in cell supernatants after 72 h by chromogenic assay.
Jeremy C. Simpson (29225)   +9 more
core   +1 more source

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