Researchers have gained new insights into physiological mechanisms that protect against blood clotting in immobilized individuals by studying animals that stay immobile for a good chunk of the year at a time: hibernating bears. “As a clinician, if you think about immobility, you always think about thrombosis,” Tobias Petzold told BioWorld. But his team’s work, which was published in the April 13, 2023, issue of Science, demonstrated that “immobility can trigger antithrombotic mechanisms.”
Base editing (BE), a technique that modifies a single nucleotide in living cells, has been successfully tested to resolve the CD3δ mutation in severe combined immunodeficiencies (SCIDs) and produce functional T cells. For now, scientists at the University of California, Los Angeles (UCLA), completed the study on patient stem cells and artificial thymic organoids, shortening the way for future clinical trials.
Janssen Pharmaceutica NV has disclosed 5-oxo-1,2,3,5,8,8a-hexahydroindolizine-3-carboxamide derivatives acting as coagulation factor XIa and plasma kallikrein (KLKB1) inhibitors reported to be useful for the treatment of thromboembolism, diabetes, diabetic retinopathy, septic shock, hereditary angioedema, arthritis, nephropathy and inflammatory disorders, among others.
Two molecules that affected the cell cycle only of acute myeloid leukemia (AML) cells could be used as a clinical strategy against this pathology. Scientists at Memorial Sloan Kettering Cancer Center and Harvard University have discovered that DEG-35 and DEG-77 arrested the cell cycle and promoted cell differentiation and apoptosis in these cells.
Current antithrombotic therapies for the prevention and management of cardiovascular disorders such as thrombosis, myocardial infarction (MI) or stroke present an associated risk of bleeding. The essential events leading to the formation of hemostatic clots are platelet activation and fibrin formation. When activated, the prostacyclin (IP) receptor prevents platelet aggregation in arteries and veins after injury.
The success of the treosulfan-based conditioning regimen in patients with β-thalassemia undergoing hematopoietic cell transplantation (HCT) is limited due to several complications, such as mixed chimerism and graft rejection. Researchers previously found that polymorphisms in the NQO1 or glutathione S-transferase A1 (GSTA1) genes had an impact on treosulfan pharmacokinetics, which then impacted related toxicities after HCT.
Fifteen years ago, at the 2008 Conference on Retroviruses and Opportunistic Infections (CROI), researchers announced that they had cured a patient – Timothy Ray Brown, initially known only as the Berlin Patient to preserve his privacy – of HIV through a hematopoietic stem cell transplant. Now, as researchers are gathered in Seattle for CROI 2023, reports of another cured patient were published Feb. 20, 2023, in Nature Medicine. Ten years after receiving a hematopoietic stem cell transplant, and 4 years after stopping antiretroviral treatment (ART), a 53-year-old patient may have been cured of HIV infection.
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