A proprietary Src homology 2 (SH2) platform of integrated technologies was applied for the discovery of REX-7117 and REX-5376, two highly potent, selective and orally available SH2 domain-targeting STAT3 inhibitors.
Researchers from Duke University presented findings from a study that aimed to assess the physiological role of Piezo1, a mechano-gated ion channel that is highly expressed in pancreatic acinar cells, in the exocrine pancreas.
Researchers from Tulane University identified NOX4, a ROS-generating enzyme, in epithelium near fibrotic foci in tissue biopsies of patients with IPF and linked its expression to epithelial cells senescence and persistence of aberrant transitional state leading to pulmonary fibrosis.
Idiopathic pulmonary fibrosis (IPF) is a disease in which several unique genes have shown expression in the lung tissue of patients with IPF. Previous findings had found the mRNA expression of band 4.1-like protein 3 (EPB41L3) to be 14-fold higher in lung fibroblasts from patients with IPF compared to control subjects.
Psoriasis is an inflammatory skin disease in which lipid metabolism is often dysregulated. ATP-citrate synthase (ACLY) is known to play a key role in lipid metabolism, but its involvement in psoriasis is not well understood.
Kinesin-like protein KIFC1 plays a role in the clustering of centrosomes in cancer cells and is being investigated in some types of cancer, concretely in melanoma.
Podocytes are a terminally differentiated cell type located in the glomerulus. Podocyte damage and the subsequent dysregulation of podocyte proteins have been implicated in various kidney disorders. Since gene delivery to podocytes using adeno associated vectors (AAVs) has been challenging due to various technological and physiological hurdles, investigators at Purespring Therapeutics Ltd. developed an AAV gene therapy platform that allowed for effective, specific and safe delivery of transgenes to podocytes.
Interstitial lung fibrotic disease (ILD) is characterized by inflammation and fibrosis of lung tissue and is associated with poor prognosis. Gat Therapeutics SL has developed GTX-011, an orally available small molecule for treating fibrotic diseases.
CTNNB1 syndrome is a rare neurodevelopmental disorder that is caused by mutations in the gene encoding β-catenin, CTNNB1, which plays a critical role in neuronal development, synapse formation and brain maturation.
About 50% of patients with small intestine neuroendocrine tumors (SI-NETs) have their prognosis negatively impacted by the development of mesenteric fibrosis, but the mechanisms behind these are poorly understood. In work at Sun Yat-sen University, single-cell RNA sequencing was performed on five primary tumor specimens and their corresponding adjacent nontumoral tissues.
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