Results 91 to 100 of about 13,000,271 (286)
Pediatric Blood &Cancer, EarlyView.ABSTRACT Purpose Vitamin K deficiency bleeding (VKDB) can occur in neonates and infants due to low placental transfer and storage of VK. Although the incidence has decreased with global VK prophylaxis at birth, VKDB remains a concern. This study aimed to assess the recent magnitude, risk factors, and outcomes of VKDB and barriers/limitations to VK ...
Nongnuch Sirachainan +16 more
wiley +1 more source
Blood, 2015 Emerging successful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk of cellular immune response to vector capsid is clearly dose dependent. To decrease the vector dose, we explored AAV-8 (
J. Crudele +8 more
semanticscholar +1 more source
Prenatal Diagnosis, EarlyView.ABSTRACT Objective Expanded carrier screening (ECS) is used to assess the reproductive probability of having an offspring affected by an autosomal recessive or X‐linked recessive genetic disorder. Rarely, a presumably healthy individual may be identified to carry variants that could influence their health.
Yan Lü +9 more
wiley +1 more source
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
Discovery of an Orally Effective Factor IX-Transferrin Fusion Protein for Hemophilia B
International Journal of Molecular Sciences, 2019 Hemophilia B is a severe blood clotting disorder caused by the deficiency of factor IX (FIX). FIX is not bioavailable when given orally due to poor stability and permeability in the gastrointestinal tract.
Chen Xie +4 more
semanticscholar +1 more source
Bleeding, Thrombosis and Vascular BiologyBackground and Aims: Intracerebral hemorrhage (ICH) is a severe and life-threatening complication in patients with hemophilia, often resulting in high mortality and long-term neurological impairment.
doaj +1 more source
Patient Related Outcome Measures, 2019 Tyler W Buckner,1 Robert Sidonio Jr,2 Michelle Witkop,3 Christine Guelcher,4 Susan Cutter,5 Neeraj N Iyer,6 David L Cooper6 1Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO, USA; 2Department of Pediatrics, Aflac ...
Buckner TW +6 more
doaj
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
Small, EarlyView.The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa +4 more
wiley +1 more source
Small, EarlyView.Inspired by the Ampullae of Lorenzini in elasmobranch fish, a biomimetic electroreceptor platform is developed using an electret layer‐coated single‐walled carbon nanotube thin‐film transistor (eSWCNT‐TFT). This platform enables non‐contact, label‐free, and real‐time monitoring of DNA encapsulation efficiency in liposome droplets by detecting net ...
Younsu Jung +11 more
wiley +1 more source
"Diagnosis of Hemophilia B Carriers, Using Taq I and Xmn I Polymorphisms of the FactorIX Gene in Iranian Individuals" [PDF]
Iranian Journal of Public Health, 2003 Hemophilia B is factor IX deficiency and is inherited as X-linked recessive disorder. The subject of carrier detection in hemophilias has received new impetus in the last several years.
P Ghandil +3 more
doaj +1 more source