Dominantly inherited mutations in the MAPT gene, which encodes the tau protein, result in a subset of tauopathies named frontotemporal lobar degeneration with tau pathology (FTLD-tau). However, the mechanisms by which MAPT mutations cause disease remain unclear. In a recent study, researchers from Washington University in St. Louis aimed to investigate the role of long non-coding RNAs (lncRNAs) and the impact of MAPT mutations on lncRNA in tauopathy.
Protein-arginine deiminase type-4 (PAD4) protein contributes to the formation of neutrophil extranuclear traps (NETs), which in turn lead to tumor growth and cancer immune escape that favors metastatic disease. The expression of PAD4 in certain types of cells, such as hematopoietic stem cells, makes the strategy of PAD4 inhibition risky due to adverse side effects.
Küleon LLC (formerly Psilosterics LLC) has announced a trifunctional serotonergic small molecule that is a full 5-HT2C receptor agonist and full antagonist of the 5-HT2A and 5-HT2B receptors with potential to treat neuropsychiatric disorders. Designated KB-128, it could be developed to treat disorders that can be modulated though 5-HT2C receptors, including schizophrenia, Alzheimer’s psychosis, depression, obesity and addiction.
Just one week after birth, the heart experiences a change in metabolism that helps it meet the high energy demand necessary to fulfill its function. This evolutionary developmental process could have medical advantages.
Recently, researchers at Cincinnati Children’s Hospital, in collaboration with colleagues in Japan, have developed a human vascular organoid model that accurately mimics the vascular damage caused by SARS-CoV-2.
Additional early-stage research and drug discovery news in brief, from: Alligator Bioscience, Athira Pharma, Coya Therapeutics, Kineta, Neurosense Therapeutics, Olatec Therapeutic, Polypid, Sola Biosciences.
Merck Sharp & Dohme Ltd. has described ceramide glucosyltransferase (glucosylceramide synthase; GLCT-1) inhibitors reported to be useful for the treatment of cancer, Lewy body dementia, diabetes, obesity, polycystic kidney disease, neurodegeneration, lysosomal storage disease and Parkinson’s disease, among others.
Shanghai Aryl Pharmtech Co. Ltd. and Zhejiang Hisun Pharmaceutical Co Ltd. have identified heterocyclic derivatives acting as Ras-related C3 botulinum toxin substrate 1 (RAC1; TC25) inhibitors reported to be useful for the treatment of melanoma, Menkes disease, rheumatoid arthritis, atherosclerosis, type 1 diabetes, Huntington’s disease and Alzheimer’s disease.
Astellas Pharma Inc. has divulged proteolysis targeting chimera (PROTAC) compounds acting as GTPase KRAS (G12D mutant) degradation inducers reported to be useful for the treatment of pancreatic cancer.
Researchers from Ascentage Pharma Group International and Ascentage Pharma (Suzhou) Co. Ltd. have synthesized tricyclic compounds acting as inhibitor of apoptosis proteins (IAP) inhibitors reported to be useful for the treatment of cancer, AIDS, autoimmune disease, immune thrombocytopenia (idiopathic thrombocytopenic purpura), infections, psoriasis, rheumatoid arthritis, and inflammatory disorders, among others.
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