Results 61 to 70 of about 12,413,959 (307)
Advanced 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
PharmaceuticalsBackground: Eptacog beta is a novel recombinant activated factor VII (rFVIIa). Preclinical studies have shown the product has a similar profile to eptacog alfa.
Jose Manuel Martin de Bustamante +5 more
doaj +1 more source
Haemophilia, 2018 Prophylaxis with replacement factor IX (FIX) reduces bleeding frequency and improves quality of life in haemophilia B patients. With prophylaxis, the likelihood of bleeding is lowered with increasing trough levels.
J. Oldenburg +9 more
semanticscholar +1 more source
Traceless Regulation of Genetic Circuitry
Advanced 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 Journal of Haemophilia Practice, 2022 In children with haemophilia (CwH), central venous access devices (CVADs) are frequently placed to aid in the delivery of factor concentrates. In those who develop inhibitors, CVADs also allow for easy venous access and facilitation of immune tolerance ...
Kwan Celia +6 more
doaj +1 more source
Haemophilia B- A coagulation disorder
Indian Journal of Clinical Anatomy and Physiology, 2022 Haemophilia is a group of inherited blood disorders in which blood does not clot properly. Bleeding disorders are due to defect in blood vessels, the coagulation mechanism or the blood platelets. When the coagulation factors are deficient, the blood does not clot properly and bleeding continues.
openaire +1 more source
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
The Journal of Haemophilia Practice, 2023 Haemophilia A and B are X-linked inherited bleeding disorders, resulting in the deficiency of clotting factor VIII and IX, respectively. Since the introduction of recombinant clotting factor concentrates in the early 1990s, the major safety concern for ...
Buckner Tyler W. +5 more
doaj +1 more source
Haemophilia, 2018 The use of musculoskeletal ultrasound (MSK‐US) following protocols for haemophilic arthropathy and the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD‐US) score can help standardize monitoring in haemophilia.
V. Jiménez‐Yuste +20 more
semanticscholar +1 more source
Annals of Neurology, EarlyView.Pathogenic DMD variants usually follow the reading‐frame rule: out‐of‐frame changes cause Duchenne muscular dystrophy, whereas in‐frame ones produce Becker muscular dystrophy (BMD). We report a 23‐year‐old man with BMD‐like weakness, calf hypertrophy, elevated creatine kinase, and dilated cardiomyopathy.
Hiroya Naruse +16 more
wiley +1 more source