Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure characterized by development of multiple fluid-filled cysts linked to increased cAMP levels and fibrosis in the kidneys leading to progressive renal failure and need for dialysis or renal transplant.
Based on findings from recent studies that have identified MUC5AC in mice infected with the nematode Trichuris trichiura and during colitis, researchers from Medical University of South Carolina aimed to assess the effects of Clostridioides difficile infection on MUC5AC in the intestinal mucosa.
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.
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