Ontology highlight
ABSTRACT:
SUBMITTER: Barbon E
PROVIDER: S-EPMC7136963 | biostudies-literature | 2020 Apr
REPOSITORIES: biostudies-literature
Barbon Elena E Ayme Gabriel G Mohamadi Amel A Ottavi Jean-François JF Kawecki Charlotte C Casari Caterina C Verhenne Sebastien S Marmier Solenne S van Wittenberghe Laetitia L Charles Severine S Collaud Fanny F Denis Cecile V CV Christophe Olivier D OD Mingozzi Federico F Lenting Peter J PJ
EMBO molecular medicine 20200311 4
Novel therapies for hemophilia, including non-factor replacement and in vivo gene therapy, are showing promising results in the clinic, including for patients having a history of inhibitor development. Here, we propose a novel therapeutic approach for hemophilia based on llama-derived single-domain antibody fragments (sdAbs) able to restore hemostasis by inhibiting the antithrombin (AT) anticoagulant pathway. We demonstrated that sdAbs engineered in multivalent conformations were able to block e ...[more]