Results 81 to 90 of about 83,381 (315)
On a break with the X: the role of repair of double-stranded DNA breaks in X-linked disease [PDF]
, 2012 The problem of managing free reactive DNA ends in eukaryotic cells has resulted in the development of a number of mechanisms in order to ensure that free ends are rendered non-reactive, or that the double-strand DNA breaks generating the free ends are ...
Cecceroni, Lucia +2 more
core +4 more sources
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
Physiological Aspects of Genetics [PDF]
, 1954 A considerable amount of evidence indicates that desoxyribonucleic acid is capable of duplicating itself, a property also possessed by genes. (By a self-duplicating material, we mean one which plays some essential role in its own production.) Watson ...
Horowitz, N. H., Owen, Ray D.
core +1 more source
Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components
Small Methods, EarlyView.The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu +6 more
wiley +1 more source
Treatment With Efmoroctocog Alfa (Elocta®) in Hemophilia: A Case Series
Clinical Management Issues, 2020 Hemophilia A is a rare X-linked disease that occurs as a result of a defect in the FVIII-encoding gene. The reduction or absence of plasma FVIII compromises the coagulation cascade, resulting in frequent bleeds, especially in joints or soft ...
Ezio Zanon +6 more
doaj +1 more source
Elucidating the etiology of idiopathic spontaneous intraperitoneal hemorrhage
Journal of Forensic Sciences, EarlyView.Abstract Free blood within the abdominal cavity (hemoperitoneum) presents a significant diagnostic and interpretive challenge. It may result from trauma or occur spontaneously in association with underlying disease conditions. When no source of fatal hemorrhage is identified, the implications extend across forensic, criminalistic, legal, and ethical ...
Dalibor Kovařík +4 more
wiley +1 more source
European Journal of Haematology, EarlyView.ABSTRACT Objectives To assess the effectiveness and safety of damoctocog alfa pegol in patients with severe and nonsevere hemophilia A in the fifth interim analysis of the ongoing HEM‐POWR study. Methods HEM‐POWR (NCT03932201) is a multinational, Phase 4, prospective observational study.
Mark T. Reding +7 more
wiley +1 more source
European Journal of Haematology, EarlyView.ABSTRACT Objectives We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom. Methods Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first‐time rVWF for the on‐demand ...
Mike Laffan +6 more
wiley +1 more source
European Journal of Haematology, EarlyView.ABSTRACT Objectives We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) receiving recombinant von Willebrand factor (rVWF) in surgical settings in the United Kingdom. Methods Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first‐time rVWF for the ...
Mike Laffan +6 more
wiley +1 more source
Association of inferior vena cava filter placement for venous thromboembolic disease and a contraindication to anticoagulation with 30-day mortality [PDF]
, 2018 Importance: Despite the absence of data from randomized clinical trials, professional societies recommend inferior vena cava (IVC) filters for patients with venous thromboembolic disease (VTE) and a contraindication to anticoagulation therapy.
Brown, David L +3 more
core +1 more source