Supplementum 293: Abstracts of the Swiss Oncology and Hematology Congress (SOHC) (Basel, Switzerland, November 19−21, 2025)
Year: 2025
Type: article
Abstract: Background and objective: Glanzmann thrombasthenia (GT) is a rare inherited bleeding disorder caused by deficiency of platelet integrin αIIbβ3, impairing platelet aggregation and clot formation.Patients present with mucocutaneous bleeding, while severe cases develop gastrointestinal (GI) and life-threatening hemorrhages.Management relies on platelet transfusions for acute episodes and recombinant factor VIIa for refractory or perioperative cases.Protein S (PS), a natural anticoagulant in blood, acts as a cofactor for activated protein C and tissue factor pathway inhibitor, and directly regulates prothrombinase and tenase complexes, thereby increasing thrombin generation (TG).We previously showed hepatic PS silencing with small interfering RNA (PS-siRNA) (Fig. A) rebalanced hemostasis in hemophilia models.This strategy may also therapeutically benefit GT.Here, we evaluated whether PS-siRNA improves TG and provides prophylactic protection against bleeding in GT.Methods: GT mice (Itgb3⁻/⁻) received a single subcutaneous dose of PS-siRNA or vehicle (Fig. B).After
Source: Swiss Medical Weekly
DOI: 10.57187/s.5128
Author: Swiss Society of Hematology
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W4416187395
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