ArticleEMBO molecular medicine2020
Antithrombin inhibition using nanobodies to correct bleeding in hemophilia.
Article in EMBO molecular medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
3 citing papers in PubMed, 2 citations in OpenAlex.
- Natural anticoagulants: A missing link in mild to moderate bleeding tendencies.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Review
- Selection and identification of single-domain antibody against Peste des Petits Ruminants virus.Journal of veterinary science · 2021Article
- Antithrombin inhibition using nanobodies to correct bleeding in hemophilia.EMBO molecular medicine · 2020Article
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
In this issue of EMBO Molecular Medicine, Barbon et al describe a new approach to rebalancing coagulation in patients with hemophilia (PWH) through targeted inhibition of anticoagulant antithrombin (AT) (Barbon et al, 2020). In contrast to previous studies that used RNA interference (RNAi) therapy to reduce AT levels (Sehgal et al, 2015; Pasi et al, 2017), the authors utilized llama-derived single-domain antibodies (sdAbs or nanobodies) to inhibit AT activity (Fig 1). These engineered sdAbs successfully restored thrombin generation in hemophilic plasma and corrected bleeding phenotype in a murine hemophilia model. Furthermore, long-term AAV8-mediated hepatic expression of the sdAb was well tolerated and associated with a sustained correction in bleeding in hemophilia A and B mice. Collectively, these exciting data uncover a novel AT-targeting approach that may be useful as an alternative therapy for restoring normal hemostasis in PWH.
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Registered trials
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