Results 71 to 80 of about 113,823 (359)

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

open access: yesAdvanced Materials, EarlyView.
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

Correlation between APTT mixing test results and inhibitor titers in patients with hemophilia A inhibitor

open access: yesZhongguo shuxue zazhi
[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]

open access: yes, 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

Traceless Regulation of Genetic Circuitry

open access: yesAdvanced Science, EarlyView.
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

open access: yesBlood Advances, 2018
: 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]

open access: yes, 2011
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

open access: yesWiener Klinische Wochenschrift, 2021
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

open access: yesAnimal 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

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

open access: yes, 2004
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?

open access: yesResearch and Practice in Thrombosis and Haemostasis, 2021
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

Home - About - Disclaimer - Privacy