Erythrocyte Binding Antigen-140 ( PfEBA-140) orthologues in human- and ape-infective Laverania species recognize Neu5Ac and Neu5Gc, but prefer ape-specific Neu5Gc. tinyurl.com/2s36m59n #glycotime #malaria

Fabry disease: a mutation in the GLA gene leading to deficient α-galactosidase A activity, resulting in lysosomal accumulation of globotriaosylceramide (Gb3). Adamantyl-GalCer inhibits the A4GALT activity, causing a decrease in Gb3 production in fibroblasts. tinyurl.com/4cb3wrrz #glycotime

@kmikolajczyk.bsky.social demonstrates small extracellular vesicles derived from Caco-2 and HEK293T cells and functionalized with Gb3 trisaccharide (Galα1→4Galβ1→4Glc) as potent neutralizers of Shiga toxins. #glycotime tinyurl.com/29y8n5yc

Can adeno-associated virus (AAV) gene therapies for hemophilia trigger tumorigenesis? Not really, as @rkaczmarekdr.bsky.social explains... tinyurl.com/3wpfsw3p #hemophilia

Where is coagulation factor VIII (FVIII) produced? After decades of research, the new data show that FVIII comes from liver sinusoidal endothelial cells located in zones 2 and 3 of hepatic lobules in mice and humans, with minimal expression in Zone 1 @rkaczmarekdr.bsky.social tinyurl.com/ydtaar2a

Last day starts with an Inspiring presentation @rkaczmarekdr.bsky.social #ISTH2025 #hemophilia #genetherapy #Toleranceinduction Will gene therapy be used for tolerance induction in future?

What percentage of hemophilia patients develop inhibitors? New data from the European Haemophilia Safety Surveillance Inhibitor reveals inhibitor rates of Hemophilia A: 4.2 per 1000 treatment years Hemophilia B: 0.1 per 1000 treatment years Read more here: www.rpthjournal.org/article/S247...

Inhibitor development in nonsevere hemophilia: data from the European Haemophilia Safety Surveillance (EUHASS) registry

Information on inhibitor development in nonsevere hemophilia and its association with clotting factor concentrate type is limited.

rpthjournal.org

Nice #glycotime review that pulls together the emerging story of glycosyltransferase specificity for cellular substrates chemistry-europe.onlinelibrary.wiley.com/doi/10.1002/...

Glycoproteins, Glycolipids, or Both: Why do Glycosyltransferases Recognize Different Acceptors?

Glycosylation is an important post-translational modification catalyzed by glycosyltransferases (GTs), which comprise a large group of enzymes with diversified specificity. Most GTs show high specifi...

chemistry-europe.onlinelibrary.wiley.com

Specificity in glycosylation! Have you ever wondered why some glycosyltransferases recognize glycoproteins, some glycolipids, and some both? Together with @kmikolajczyk.bsky.social and @rkaczmarekdr.bsky.social, we try to answer that complicated question. #glycotime tinyurl.com/5b6kxhp3

It is essential for #genetherapy progress for unsuccessful clinicals trials to be published. We discuss recent study showing no benefit for prophylactic steroids after #AAV for #hemophilia. authors.elsevier.com/a/1k-re5TuFx... w/ @rkaczmarekdr.bsky.social

Comprehensive review of gene therapy in #hemophilia accepted for publication by Molecular Therapy. The pre-proof is on line at tinyurl.com/3sb4nuex

Our paper on inhibitor trends is now published in the journal Haemophilia. The number of inhibitors in previously untreated patients has markedly reduced, but it is possible that this is due to delayed exposure to FVIII. tinyurl.com/2mr3t5e3

I was pleased to moderate the session on basic and translational research in coagulation and fibrinolysis with Dr. Carlos Bravo-Perez at #ASH2024 earlier today. Six excellent presentations are now available on the Virtual Platform.