Undiagnosed bleeding disorders put people at risk due to bleeding without an optimal treatment strategy. Spanish researchers from the Hospital Universitario La Paz and Universidad Autónoma de Madrid have focused on the targeting of tissue factor pathway inhibitor (TFPI) as a potential approach for this medical need by using TFPI blocker antibodies.
Gray platelet syndrome is an autosomal recessive platelet disorder characterized by macrothrombocytopenia and deficiency or decreased levels of alpha granules that confer a grayish appearance to the platelets. The genetic cause is located at chromosome locus 3p21, affecting the NBEAL2 gene.
Previous research has suggested that factor VIII (FVIII) can regulate the osteoprotegerin (OPG)/RANKL system, which appears to play a role in hemophilic arthropathy. Investigators have now aimed to measure the OPG levels in patients with hemophilia A/B and assess their correlation with the levels of FVIII/FIX.
Glanzmann thrombasthenia (GT) is a rare bleeding disorder caused by defects in the expression of platelet surface integrins, such as integrin alpha-IIb (GPIIb, encoded by ITGA2B).
The combination of two sequencing techniques has unveiled features of a subpopulation of cells that could be producing plaques in atherosclerosis. This process is associated with an autoimmune component driven by CD4+ T cells, according to a study from researchers at Leiden University.
“I think that we can clearly say now that atherosclerosis is a very clear autoimmune component. It is a multifactorial disease, a combination of genes, and lifestyle, but also inflammation and the immune system,” Ilze Bot and Bram Slütter, associate professors at the Division of Biotherapeutics of Leiden University, told BioWorld.
Sickle cell disease (SCD) is autosomal recessive disorder caused by mutations in the β-globin gene, and induction of fetal γ-globin is considered an established therapeutic strategy for the treatment of this disease. A research team led by scientists at Kyorin Pharmaceutical Co. Ltd. has discovered RK-701, a small-molecule inhibitor of G9a and G9a-like protein (GLP) as a potential therapeutic agent for SCD.
A genome-wide association study (GWAS) from The University of Queensland has linked blood cell traits (BCTs) and neurological and psychiatric disorders (NPDs), providing a tool to improve patient treatments or repurposing different drugs. The researchers also found a cause-effect relationship between Parkinson's and platelet distribution width. In their study, published Jan. 25, 2023, in Cell Genomics, the scientists observed the genetic overlap between common NPDs and 29 BCTs, including functional genes, regulatory elements and new genetic correlations linked to hematological data and for these diseases.
Mabwell Therapeutics Inc., a wholly owned subsidiary of Mabwell (Shanghai) Bioscience Co. Ltd., has entered into a license agreement with Disc Medicine Inc. for 9MW3011 (MWTX-001, MWTX-002 & MWTX-003) for hematologic diseases.
By combining drug sensitivity with genomic profiling of tumor cells, a study from St. Jude Children's Research Hospital with more than 800 patients has shown a wide diversity in drug sensitivity for pediatric acute lymphoblastic leukemia (ALL) and defined six patterns of response to treatment. “This work provides a framework for ‘functional precision medicine’,” corresponding author Jun Yang, vice chair of the Department of Pharmacy and Pharmaceutical Sciences at St. Jude Children's Research Hospital, told BioWorld.
Scientists based at Beijing Institute of Radiation Medicine have reported preclinical evaluation of a novel recombinant activated human factor VII (rFVIIa), GEN-0828, being developed as potential candidate for the treatment of hemophilia and trauma hemorrhage. GEN-0828 is rFVIIa that was mainly produced from Chinese hamster ovary (CHO) cells.
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