Matthew Stephen Wake, Anaïs Palin, Audrey Belot, Mathieu Berger, Megane Lorgouilloux, Margot Bichon, Jonathan Papworth, Luke Thomas Bayliss, Ben Grimshaw, Natalie Rynkiewicz, Jemima Paterson, Alicia Poindron, Erin Spearing, Emily Carter, Robyne Hudson, Millie Campbell, Verena Petzer, Céline Besson-Fournier, Chloé Latour, Amélie Largounez, Ophélie Goubeyre, Alexis Fay, Helene Coppin, Marie-Paule Roth, Igor Theurl, Volker Germaschewski, Delphine Meynard
Iron plays a major role in the deterioration of β-thalassemia. Indeed, the high levels of transferrin saturation and iron delivered to erythroid progenitors are associated with production of α-globin precipitates that negatively affect erythropoiesis. Matriptase-2/TMPRSS6, a membrane-bound serine protease expressed in hepatocytes, negatively modulates hepcidin production and thus is a key target to prevent iron overload in β-thalassemia. To address safety concerns raised by the suppression of Tmprss6 by anti-sense oligonucleotides or small interfering RNA, we tested a fully human anti-matriptase-2 antibody, RLYB331, that blocks the protease activity of matriptase-2...
January 19, 2024: Blood Advances