Cellarity Inc. has divulged DCN1-like protein 1 (DCUN1D1; RP42) and/or DCN2 inhibitors reported to be useful for treatment of sickle cell and thalassemia disorder.
Scientists at the Center for Genomic Regulation (CRG) have developed an AI-based tool to design thousands of sequences that regulate DNA. They have also synthesized these molecules, called enhancers, to control gene activation in mouse hematopoietic stem cells, which they have tested in vitro.
Anticoagulant drugs, whether classical ones such as warfarin and heparin or newer ones such as dabigatran and apixaban, can be effective for treating and preventing deep vein thrombosis, myocardial infarction, ischemic stroke and pulmonary embolisms. Targeting factor XIa, a serine protease in the coagulation pathway, may inhibit thrombosis without increasing bleeding risk. Numerous inhibitors of factor XIa have been developed and several have entered clinical trials, but most have shown problems of poor efficacy, inadequate selectivity or drug-drug interactions.
A recent study by researchers from Texas A&M University presented a new vaccine designed to target the ligand-binding domain of the serotonin 2A receptor (5-HT2AR), which resides in the second extracellular loop (EL2) and was previously identified as the key region for receptor activation. The new candidate, called EL2-5HTVac, was shown to provide a long-lasting and selective therapeutic approach to avoid increased bleeding risk complications.
The University of Michigan has divulged lysine-specific histone demethylase 1A (KDM1A; LSD1) inhibitors reported to be useful for the treatment of cancer, autism, myocardial fibrosis and more.
Sepsis-induced coagulopathy (SIC) is a complication of sepsis tied to high mortality in patients. Anticoagulation using a coagulation factor IIa and Xa dual inhibitor might have the potential to improve the treatment of this severe condition.
Two simultaneous but independent studies published in Science identified, by introducing mutants into its genome, the essential and nonessential genes of Plasmodium knowlesi, one of the malaria parasites related to the dreaded Plasmodium vivax. Their results could help in the development and prioritization of antimalarial strategies.
Mouse models for hemophilia A are commonly generated by factor VIII (FVIII) knockout, however, these mice rapidly develop anti-FVIII antibodies during repetitive FVIII administration. To overcome this preclinical research challenge, investigators from Octapharma AG and affiliated organizations developed a new model unable to produce antibodies but otherwise not immunosuppressed.
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