Results 31 to 40 of about 48,006 (264)
Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy
Molecular Therapy: Methods & Clinical Development, 2019 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.
Benjamin J. Samelson-Jones +1 more
doaj +1 more source
Haemophilia A: pharmacoeconomic review of prophylaxis treatment versus on-demand [PDF]
, 2015 Objectives. Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis.
BOCCIA, Antonio +4 more
core +2 more sources
Construction of pathogenic Sec16a mutation mouse model using CRISPR/Cas9
Animal Models and Experimental Medicine, EarlyView.Yaqiang Hu et al. engineered a pathogenic Sec16a mutant mouse model using CRISPR/Cas9 technology. They observed that the Sec16a mutant mice displayed diminished learning and memory capabilities, along with a limb‐clasping phenotype upon tail suspension.
Yaqiang Hu +6 more
wiley +1 more source
PLoS ONE, 2020 IntroductionThe rapid clearance of factor IX necessitates frequent intravenous administrations to achieve effective prophylaxis for patients with hemophilia B.
Timothy C Nichols +4 more
doaj +1 more source
Queen Victoria and The Royal Disease [PDF]
, 2018 Queen Victoria, Queen of England and Ireland and Empress of India, is the second longest ruling monarch of England, she sat on the throne for sixty –three years and was so crucial to the formation of England that there is a whole era names after her, The
Mullen, Madeline
core +1 more source
Biotechnology and Bioengineering, EarlyView.ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai +9 more
wiley +1 more source
Hematology ReportsBackground/Objectives: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of ...
Aida M. S. Salem +6 more
doaj +1 more source
A Mechanistic Model of rAAV Production in Synthetic Cell Lines
Biotechnology and Bioengineering, EarlyView.ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan +5 more
wiley +1 more source
A Case of Acquired Hemophilia A and Congenital Hemophilia B
Cureus, 2022 Congenital hemophilia B is a rare, inherited X-linked bleeding disorder caused by a deficiency of factor IX (FIX). Acquired hemophilia A is a rare, acquired bleeding disorder which presents as new onset bleeding in older adults due to the development of autoantibodies against factor VIII (FVIII).
Fortier, Julia C +4 more
openaire +2 more sources
Diagnosis of von Willebrand disease in Argentina: a single institution experience [PDF]
, 2017 von Willebrand disease (VWD) is the most common autosomal bleeding disorder, mostlyinherited as dominant trait. VWD is due to deficiency/abnormality of von Willebrand factor (VWF).
Blanco, Alicia Noemi +5 more
core +1 more source