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Endothelial-specific knockout of the scramblase TMEM16F impairs in vivo clot formation.
Shock
January 2025
Pharmacology, University of Vermont, Burlington, VT.
Objective: Loss of function of the phospholipid scramblase (PLS) TMEM16F results in Scott Syndrome, a hereditary bleeding disorder generally attributed to intrinsic platelet dysfunction. The role of TMEM16F in endothelial cells, however, is not well understood. We sought to test the hypothesis that endothelial TMEM16F contributes to hemostasis by measuring bleeding time and venous clotting in endothelial-specific knockout (ECKO) mice.
View Article and Find Full Text PDFOperationalising a Haemophilia Gene Editing Lexicon for Practical Use.
Haemophilia
January 2025
Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
Introduction: Gene editing therapies offer the possibility of substantial improvement in treatment and quality of life for people with haemophilia (PWH) in a landscape of dynamic therapeutic advancement. Developing a common and understandable language to discuss gene editing will be essential to ensure these treatments can be deployed in a safe and effective manner with fully informed and shared decision-making between healthcare professionals (HCPs) and PWH. A lexicon explaining and clarifying key concepts is one potential tool to address these aims.
View Article and Find Full Text PDFGene-ius at work: Hemophilia B treatment enters a new era.
Am J Health Syst Pharm
January 2025
Department of Pharmacy Practice, Auburn University Harrison College of Pharmacy, Auburn, AL, USA.
Disclaimer: In an effort to expedite the publication of articles, AJHP is posting manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time.
View Article and Find Full Text PDFAortoiliac arterial thrombosis and renal artery stenosis in a patient with neonatal multisystem inflammatory syndrome: a case report and review of literature.
Front Pediatr
January 2025
Division of Neonatology, Department of Pediatrics, Faculty of Medicine Vajira Hospital, Navamindradhiraj University, Bangkok, Thailand.
Background: Multisystem inflammatory syndrome in neonates (MIS-N) is a rare condition thought to be associated with prenatal exposure to maternal severe acute respiratory syndrome coronavirus 2 infection. This immune-mediated hyperinflammation has been described in neonates with multiorgan dysfunction, including cardiopulmonary, encephalopathy, coagulopathy, and vascular complications. However, renovascular complications in MIS-N are rare.
View Article and Find Full Text PDFSafety and Outcomes of Percutaneous Dilatational Tracheostomy in Patients with Hematologic Malignancies: A Retrospective Cohort Study.
J Clin Med
January 2025
Ruth and Bruce Rappaport Faculty of Medicine, Technion, 1 Efron St. Bat Galim, Haifa 3525433, Israel.
: Patients with hematologic malignancy (HM) often experience high rates of thrombocytopenia, thrombocytopathy, anemia, leukopenia, and coagulopathy, which can significantly increase the risk of procedural and postoperative complications. This study aimed to evaluate the safety and outcomes of percutaneous dilatational tracheostomy (PDT) in critically ill patients with HM. : This retrospective cohort study included patients with HM who underwent PDT between 2012 and 2023 at a tertiary academic center.
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