Tau is an intrinsically disordered protein that regulates the stability and dynamics of microtubules in physiological conditions. Recent work has revealed the involvement of tau in various neuronal processes. Researchers from the University of California and collaborators aimed to systematically investigate the cellular factors that control the accumulation of tau aggregates in human neurons.
Opioid use disorder (OUD) causes high morbidity and mortality rates, with fentanyl driving unprecedented overdose rates. Researchers from the University of California have used an AI-based drug discovery platform with the aim of identifying preclinical drug candidates for OUD.
The University of California has synthesized proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors reported to be useful for the treatment of cancer, atherosclerosis and stroke.
The University of California has synthesized α-synuclein (SNCA) and/or amyloid-β protein and/or microtubule-associated protein tau (PHF-tau; MAPT) propagation inhibitors reported to be useful for diagnosis and treatment of multiple system atrophy, Parkinson’s disease and Alzheimer’s disease.
Autosomal dominant polycystic kidney disease (PKD) is a chronic and debilitating condition affecting over 12 million patients worldwide. PKD arises from mutations in the polycystin-1 (PC1) or PC2 genes. PKD is characterized by the formation of fluid-filled cysts in the kidneys, leading to inflammation, fibrosis and organ damage, ultimately resulting in renal failure.
Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by synaptic dysfunction, neuronal loss and the accumulation of amyloid plaques and neurofibrillary tangles, ultimately leading to cognitive decline. Despite significant research efforts, no existing treatment has proven effective enough to stop or reverse the progression of the disease.
The third most significant genetic risk factor for late-onset Alzheimer’s disease is a mutation in the clusterin (CLU) gene that disrupts the expression of sCLU, a cytoprotective protein involved in preventing protein aggregation and promoting the clearance of misfolded proteins.
Although CAR T-cell therapies have reached significant clinical success in hematological malignancies, their utility in solid tumors remains limited. One of the main challenges is the scarcity of truly cancer-specific antigens for precise targeting of solid tumors. The use of engineered small, specific antigen-binding domains, such as nanobodies, could be a potential strategy to improve the specificity and efficacy of CAR T cells against solid tumors.
University of California has described tropane derivatives acting as 5-HT2 and muscarinic receptor ligands reported to be useful for the treatment of psychiatric disorders, depression, neurodegeneration, substance abuse and dependence, migraine, Alzheimer’s disease and Parkinson’s disease.
