Results 31 to 40 of about 50,045 (313)
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
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
A Case of Heel Reconstruction with a Reverse Sural Artery Flap in a Hemophilia B Patient [PDF]
Archives of Plastic Surgery, 2012 Hemophilia B is a rare blood coagulation disorder. Complications such as bleeding and hematoma can cause necrosis of flaps, wound disruption, and the disturbance of wound healing.
Byung Kwon Lee, Jeong Su Shim
doaj +2 more sources
Immune tolerance induction in a severe hemophilia B child with low titer inhibitors
Journal of Applied Hematology, 2022 Hemophilia B is an X-linked inherited bleeding disorder caused by either the absence or reduced biosynthesis of clotting factor IX (FIX). This disorder affects approximately 1 in 30,000 male individuals worldwide. Patients with severe form (FIX
Asia Almulla, Najam Awan, Faisl Khanani
doaj +1 more source
, 2017 Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core +2 more sources
Advanced Functional Materials, EarlyView.Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley +12 more
wiley +1 more source
Cardiac surgery in patients with Hemophilia:is it safe?
Journal of Cardiothoracic Surgery, 2020 Background The life expectancy of hemophiliacs is similar to that of the general population. As a result, the prevalence of age-related cardiovascular diseases has increased.
Amjad Shalabi +9 more
doaj +1 more source
BMC Surgery, 2022 Background Hemophilia is a recessive hemorrhagic disease relevant to X chromosome. In mild hemophilia cases, spontaneous bleeding is rare and the blood clotting function is normal, but severe bleeding may occur after trauma or surgery.
Xiaoying Jiang +4 more
doaj +1 more source
In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]
, 2018 Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana +6 more
core +1 more source
Advanced Healthcare Materials, EarlyView.This study presents guanidinium‐ and indole‐functionalized polyphosphoesters as degradable, non‐viral gene delivery vectors. Through precise tuning of charge density and hydrophobicity, these polymers form stable polyplexes with low toxicity. Remarkably, minor structural changes yield up to 200‐fold differences in transfection efficiency, highlighting ...
Markus Kötzsche +8 more
wiley +1 more source