Scientists at Medical University of Vienna, The University of Queensland and Universität Innsbruck have identified cysteine stapled dynorphin A (CSD) analogues acting as κ-opioid receptor antagonists reported to be useful for the treatment of anxiety disorders, depression, substance abuse and dependence, and stress disorders.
Pluvia AS has synthesized pharmacological chaperones able to stabilize phenylalanine hydroxylase (PAH) reported to be useful for the treatment of hyperphenylalaninemia, particularly phenylketonuria (PKU).
National Institute of Biological Sciences has disclosed pyrazole urea derivatives acting as inhibitor of nuclear factor κB kinase subunit ε (IKBKE; IKK-i) and/or TANK-binding kinase 1 (TBK1; NAK; T2K) inhibitors reported to be useful for the treatment of cancer, diabetes, obesity and inflammatory disorders.
The estrogen receptor-positive (ER+) subtype accounts for 70% of all mammary carcinoma (MC) cases. ER+ MC patients display a prolonged clinical dormancy state, with a 70% recurrence 5-20 years after clinical remission and high mortality.
Previous studies revealed a positive correlation between O-GlcNAcylation and tumor growth via the stabilization of target proteins, with O-GlcNAcylation transferase (OGT) being the only enzyme capable of catalyzing the addition of O-GlcNAcylation to these proteins. Scientists at Sun Yat-Sen University and affiliated organizations investigated the role of OGT in the progression and drug resistance of multiple myeloma.
Inclusion body myositis (IBM) is an inflammatory myopathy causing proximal and distal muscle weakness. IBM’s cause remains unknown, lacking validated models, biomarkers and effective treatment strategies. Histopathological studies identified inflammatory infiltrates, rimmed vacuoles, and mitochondrial changes in the muscles of IBM patients.
Inflammatory bowel disease (IBD) is impacted by genetic, environmental and immunological factors, where the imbalance in T-cell immune responses significantly promotes its progression. In recent years, the role of RNA modifications in epigenetic regulation has caught significant attention in research; among these, N4-acetylcytidine (ac4C) is the only acetylation process in RNA and plays a role in several biological processes, but its implication in the functioning of immune cells is largely unknown to date.
Iqure Pharma Inc. has received institutional review board (IRB) approval for a first-in-human study of iQ-007 in healthy volunteers. This follows recent formal approval from the Human Research Ethics Committee (HREC) in Australia for the phase I study, which is expected to begin next month.
The Ca2+ stored in the cellular endoplasmic reticulum (ER) plays a crucial role in protein folding and lipid transfer, and its impairment leads to cellular ER stress. When chronic cellular ER stress occurs in the liver, it triggers the pathogenesis of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH). Previous reports found that NACHT and WD repeat domain containing 1 (NWD1) localized in the ER and mitochondria in neural stem/progenitor cells, but the significance of NWD1 outside the brain is not well known.
Although safe and effective vaccines for SARS-CoV-2 have been successfully developed, there are currently no therapeutic approaches available for treating acute infection, particularly for individuals at high risk of severe disease progression, and for preparedness against a potential new coronavirus pandemic.