The I148M mutation in the PNPLA3 gene, which encodes patatin-like phospholipase domain-containing protein 3, is known to confer risk of fatty liver, cirrhosis and hepatic inflammation, which may lead to hepatocellular carcinoma or metabolic dysfunction-associated steatohepatitis (MASH).
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
Systemic Bio LLC has developed a 3D bioprinted human Vascularized Integrated Organ System (h-VIOS) platform that better recapitulates the native-like liver architecture and vascularization, integrating PHHs and non-parenchymal cells.
Pannexin 1 (PANX1) forms channels that may release signaling metabolites that are involved in a variety of pathophysiological processes, such as asthma, diabetes, hypertension or inflammatory bowel disease (IBD), among others. Inhibition of PANX1 when dysregulated has been proposed as a therapeutic approach in the treatment of IBD.
Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases.
Scientists at Massachusetts General Hospital have revealed how chronic liver injury alters hepatic stellate cells (HSCs), triggering the formation of fibrotic scarring in the liver. The researchers have shown that this cell type transdifferentiates into myofibroblasts, which generate excess extracellular matrix, driven by the activation of ABHD17B, an enzyme that could be investigated as a therapeutic target to inhibit liver fibrosis.
Fibroblast activation protein (FAP) is a serine protease, the expression of which increases with pathogenic fibroblasts in the fibrotic liver during metabolic dysfunction-associated steatohepatitis (MASH) and might induce fibrosis by cleaving several proteins that regulate extracellular matrix turnover and metabolism, including α2-antiplasmin (α2-AP) and fibroblast growth factor 21 (FGF21). Astrazeneca plc recently presented new results on their research regarding their oral small-molecule FAP inhibitor, AZD-2389, as a candidate drug for treating MASH.
Research published online in Nature on March 19, 2025, closely examines the changes occurring in the gastric epithelium during the progression toward cancer development. Certain mutations that occur in normal, nonreproductive cells over time can make these cells more prone to becoming cancerous later. The project began as a collaboration between the labs of Mike Stratton at the Sanger Institute and Suet Yi Leung from the University of Hong Kong, funded by the Wellcome Trust and the Kadoorie Charitable Foundation.
Researchers from Chinese Academy of Sciences detailed the creation of a new dextran sulfate sodium (DSS)-based mouse model of immune checkpoint blockade (ICB) therapy-associated colitis.
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