Results 71 to 80 of about 113,823 (359)
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode +16 more
wiley +1 more source
[Objective] To study the correlation between activated partial thromboplastin time (APTT) mixing test results and the inhibitor titers in hemophilia A inhibitor-positive patients.
WANG Tiantian +8 more
doaj +1 more source
Queen Victoria and The Royal Disease [PDF]
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
Traceless Regulation of Genetic Circuitry
Energy‐based, as opposed to molecular, control offers unprecedented improvements in key circuit parameters. This review summarizes the fundamentals of such traceless switches, categorizes them by trigger modalities, and compares and contrasts distinct advantages as well as shortcomings of each kind.
Gokberk Unal, Martin Fussenegger
wiley +1 more source
The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B
: Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie +4 more
doaj +1 more source
Haemophilia and joint disease: pathophysiology, evaluation and management [PDF]
In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles.
Berntorp, Erik, Knobe, Karin
core +3 more sources
Pain management in hemophilia: expert recommendations
As a typical consequence of bleeding into muscles and joints, patients with severe hemophilia suffer from acute and chronic pain. In spite of its high prevalence, pain in this patient group is not always sufficiently considered or treated in an effective
W. Stromer +11 more
semanticscholar +1 more source
Construction of pathogenic Sec16a mutation mouse model using CRISPR/Cas9
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
Progress in the use of adeno-associated viral vectors for gene therapy [PDF]
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core +1 more source
Living with a “hemophilia‐free mind” – The new ambition of hemophilia care?
Despite the numerous and groundbreaking therapeutic advances made in the field of hemophilia over the past decades and particularly in recent years, hemophilia remains a disease that has a major impact on the daily lives of our patients, through the ...
Evelien Krumb, C. Hermans
semanticscholar +1 more source

